john m. ravits, md - uc san diego school of medicine€¦ · · 2017-07-20american board of...

CURRICULUM VITAE

John M. Ravits, MD

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December 16, 2015

PERSONAL INFORMATION

Date/Place of Birth April 7, 1953, St. Paul, MN, USA Address University of California, San Diego

9500 Gilman Drive, Mail Code: 0624 La Jolla, CA 92093-0670 Phone Number 858-246-1154 Email [email protected] Citizenship USA

EDUCATION AND TRAINING Institute and Location Degree Year Field of Study Yale University, New Haven, CT B.A. 1975 English Mayo Medical School, Rochester, MN M.D. 1979 Medicine University of California, San Diego, CA 1979-80 Intern in Medicine 1980-83 Resident in Neurology Harvard Medical School, Brigham and

Women’s Hospital, Boston, MA 1983-84 Clinical Research Fellow

in Neurophysiology National Institutes of Health, National

Institute of Neurological Disorders and Stroke, Bethesda, MD

1984-86 Medical Staff Fellow in Clinical Neurophysiology

Rady Health Sciences Leadership Academy, University of California, San Diego, San Diego, CA

Certificate 2012-13 Health Sciences Leadership

POSITIONS HELD

Institution and Location Position and title Dates Virginia Mason Medical Center, Neurology

Section, Seattle, WA Associate Staff 1986-88

Medical Staff 1988-2011 Director of Clinical Neurophysiology 1986-2011 Section Head 1987-2004 Benaroya Research Institute, Seattle, WA Affiliate Investigator 2004-07 Research Associate Member 2007-11 University of Washington, Seattle, WA Clinical Instructor (Medicine) 1987-89 Clinical Assistant Professor

(Medicine) 1989-96

Clinical Associate Professor (Neurology)

1996-2007

Clinical Professor (Neurology) 2007-2011 University of California, San Diego,

Department of Neurosciences, La Jolla, CA

Professor of Clinical Neurosciences 2011-present

Director, ALS Programs 2011-present International Mentor, Health

Sciences International 2013-present

Vice Chair, Adult Neurology and the Neurological Sciences

2014-present

John M Ravits M.D. CV (cont) rev July 16, 2017

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BOARD CERTIFICATIONS Board Organization Area Date Certificate National Board of Medical Examiners Medicine 1980 #223505 American Board of Psychiatry and Neurology Neurology 1985 #27722 American Board of Electrodiagnostic Medicine Electrodiagnosis 1989 #718 American Board of Psychiatry and Neurology Clinical

Neurophysiology 1994 & 2004

#156

American Board of Psychiatry and Neurology Neuromuscular Medicine

2008 #157

PROFESSIONAL SOCIETY MEMBERSHIPS (Active): Society Status Date American Academy of Neurology Member 1992 Fellow 2003 American Association of Neuromuscular and Electrodiagnostic Medicine

Member

1984

Fellow 1986 American Neurologic Association Member 2010 Fellow 2011 World Federation of Neurology Member 1998 Society for Neurosciences Member 2006

HONORS Honor Dates Aurelian Honor Society, Yale University 1974-5 Commencement Speaker, Mayo Medical School 1979 Student Teaching Award, University of California, San Diego 1981 King County’s “Best Doctors”, Seattle Magazine 2000, 2005 Puget Sound “Top Doctors”, Consumers’ CHECKBOOK 2002, 2003, 2006 Spirit of Lou Gehrig Award, Seattle ALS Association (Presented at Safeco Field

on Lou Gehrig’s 100th Birthday and threw out the 1st pitch) 2003, 2011

Golden Stethoscope Award for top doctors, Seattle Metropolitan Magazine 2008 University of Washington Alpha Omega Alpha Honor Medical Society’s Volunteer Clinical Faculty of the Year Award

2009

Best Doctors in America, Best Doctors 2010-present Seattle Super Doctors (top 5% of Seattle’s doctors), SuperDoctors.com 2011 ALS Champion, Greater San Diego ALS Association Chapter 2013 University of California, San Diego, Senior Faculty Teaching Award (Neurology) 2012-3, 2013-4 San Diego’s Best Doctors, SD Metro Magazine 2015-16 US Top Doctors, Castle Connolly 2015-16 Sheila Essey Award, American Academy of Neurology and ALS Association 2017

ACADEMIC ACTIVITIES

Activity and Organization Role Time Period Advisory Board: World Federation of Neurology, Research Subcommittee on ALS/MND

Member

2010-present

Journal Boards: Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

Editorial Board

2006-present


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J Neurodegeneration and Regeneration Editorial Board 2004-10 Translational Research Consortia: Western ALS (WALS) Study Group Member

Co-Chair Chair

1998-present 2012-2015 2015-present

Northeast ALS (NEALS) Study Group Director of Regional Site

2011-2016

Grant Reviewing (Standing): NINDS R13 Special Emphasis Panel

Member

2016-present

Muscular Dystrophy Association: Medical Advisory Committee

Member

2008

Scientific Advisory Committee Member 2009-present ALS Association Scientific Research Grant Committee Member 2014-present Department of Defense and US Army Medical Research

Congressionally Directed Medical Research Program on ALS

Member 2015-present

Grant Reviewing (Ad Hoc): Canada: ALS Society of Canada

Reviewer

2009, 2010, 2014

France: Thierry Latran Foundation Reviewer 2010, 2013 Ireland: SFI-Pfizer Biotherapeutics Innovation Award

Programme Reviewer 2014

Italy: AriSLA Reviewer 2010, 2014 Switzerland: Swiss National Science Foundation Reviewer 2013 UK: Motor Neurone Disease Association Reviewer 2009, 2010,

2015 US: NINDS Special Emphasis Panels SMA Reviewer 2011 NINDS ZRG1 MDCN-A(03) Reviewer 2014 NINDS ZNS1 SRB-E (58) (Clinical Trials Methods Course) Reviewer 2014 NINDS ZTR1 CI-4 (CTSA Collaborative Innovation Award Review U01 and R21)

Reviewer 2017

American Brain Foundation of the AAN and ALS Association Joint Review Committee for the Richard Olney, MD, Clinician-Scientist Development Award

Reviewer, Clinical Research Training Fellowship

2012-present

Medical Research Council, UK Reviewer 2016

Journal Reviewing (Ad Hoc):

Muscle & Nerve Reviewer 2011-present Journal of Clinical Neurophysiology Reviewer 2010, 2011 Journal of Neurological Science Reviewer 2010, 2011 Acta Neurologica Scandinavia Reviewer 2010 Clinical Genetics Reviewer 2012 Annals of Neurology Reviewer 2012, 2014,

2015 Neurology Reviewer 2013-present Brain Research Reviewer 2012. 2013


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Drug Discovery Today Reviewer 2013 Rare Diseases Reviewer 2014 Neurotherapeutics Reviewer 2014 Human Molecular Genetics Reviewer 2015 Advanced Drug Delivery Reviewer 2015 Cerebral Cortex Reviewer 2015 Journal of Cell Biology Reviewer 2015 Science Translational Medicine Reviewer 2015 Acta Neuropathologica Reviewer 2015 Cellular and Molecular Neurobiology Reviewer 2015 Journal Neurology, Neurophysiology, and Psychiatry Reviewer 2016 Mediators of Inflammation Reviewer 2017 Meetings and Symposia Organized Symposium on Stem Cell Approaches to ALS Therapy, Stanford Consortium of Regenerative Medicine, La Jolla, CA. May, 2014. Funding supported by California Institute of Regenerative Medicine.

Co-Chair

2014

International ALS and MND Symposium, Dec 9-13 , Orlando, FL

Program Committee

2015

California Research Summit, Jan 8-9, 2016, San Diego, CA Co-Chair 2016

Propagation in Neurodegeneration, Aug 8-11, 2016, Dublin Ireland (Zing Conference)

Co-Chair 2016

Stem Cell Therapies in ALS, World Federation of Neurology Satellite Symposium, Dec 6, 2016, Dublin Ireland

Chair 2016

COMMITTEES, OFFICES AND ORGANIZATIONAL ACTIVITIES Institution Activity Time Period Virginia Mason Medical Center Hyperbaric Committee 1988-93 Insurance Committee 1990-96 Personnel 1990-94 Referral Management 1994-95 Head, Section of Neurology 1996-02 Clinical Research Chair 2005-06 Clinical Research Chair 2007-08 Puget Sound Neurologic

Association President 1995-96

Neurology Advisory Board for ALS C.A.R.E.

1995-1999

American Association of Electrodiagnostic Medicine

Journal Committee 1988

Course Committee 1989-90 Constitution Committee 1991-92 Equipment & Computer Program 1994-95 Program Committee 1995-98 Professional Practices Committee 1999-02 AAEM Washington State Liaison 19998-02


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American Academy of Neurology Strategic Direction #5 Task Force on New Tools for Neurologists

1997-99

American Board of Psychiatry and Neurology

Board Examiner 1989, 1993, 1996, 2001, 2003, 2004

American Board of Electro-diagnostic Medicine

Board Examiner 1997,2000, 2001

University of California, San Diego Head, Neuromuscular Diseases 2012-present Neurodegenerations Integration Group 2012-2014 King Fahad Specialist Hospital’s Collab,

Dammam, Saudi Arabia 2012-present

Residency and Fellowship Committee 2011-present Neurosciences Resource Committee 2012-present Neurosciences Executive Council 2013-present Neurosciences Academic Review

Committees (annual ad hoc committees) 2012-present

International Mentor, Department of Health Sciences International

2013-present

UCSD Lab Utilization Committee 2015-present UCSD Human Anatomical Specimen and

Oversight Committee (HASTOC) 2016-present

Neurosciences Search Committees: Epilepsy (Chair) General Neurology (Member) Neuromuscular (Chair)

2015-16 2016-preent 2015-present

LICENSURE

California (active) Washington (inactive) Massachusetts (inactive) Virginia (inactive)

CLINICAL SUBSPECIALTY INTERESTS: Neuromuscular diseases especially ALS and motor neuron diseases and immune-mediated diseases; clinical neurophysiology and electrodiagnosis.

RESEARCH INTERESTS Propagation of neurodegeneration—phenotype, neuropathology, mechanisms ALS and functional genomics, laser capture microdissection and cell-specific gene expression,

bioinformatics, exon splicing, ALS molecular neuropathology, and tissue banking C9orf72-ALS/FTD molecular pathology and genomic mechanisms Antisense oligonucleotide therapy ALS biomarkers ALS clinical trials

PERSONAL STATEMENT I am a physician-scientist at University of California, San Diego (UCSD) specializing in amyotrophic lateral sclerosis (ALS). My formal training is in clinical neurology, clinical neurophysiology and neuromuscular diseases, and I am board-certified in all three areas. I currently direct UCSD’s ALS Center of Excellence, which includes an active clinic program in diagnosis and a Multidisciplinary


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Care Program following a panel of about 100 patients. I direct UCSD’s ALS Translational Research Program, which is active in observational studies and biobanking, including NIH-funded U54 Rare Disease Consortium, ALS Association funded biorepository, Target ALS funded neuropathology tissue bank, and Biogen, Inc funded Methodology Study. The ALS Translational Research Program also carries out clinical trials, and is involved in the international effort to develop antisense oligonucleotide therapies for ALS in an effort involving academia and industry and beginning with SOD1-mutation patients. My primary research interest is neuroanatomical propagation of ALS and this has defined my research direction at the clinical, neuropathological and experimental level characterizing, understanding, and modeling onset and progression neuroanatomically. Related to this, I have tissue repositories from phenotypically well-characterized patients and use to study disease neuropathologically and using functionally genomics in motor neurons using laser microdissection. Another major effort of my research laboratory is studying repeat expanded C9orf72-ALS, trying to tease apart the contributions from sense strand and antisense strand, and to identify optimal targets for antisense oligonucleotides.

SYNOPSIS OF MAJOR CONTRIBUTIONS TO SCIENCE AND MEDICINE

1. Neuroanatomic propagation: My primary contribution to the ALS field has been to characterize, and model ALS as a disease that progresses neuro-anatomically as well as temporally. In a series of papers, I deconstructed ALS phenotypes to show that in addition to the well-appreciated involvement of motor neurons, important aspects of ALS biology are neuroanatomical: focality of the onset, random location, variably distribution between upper and lower motor neurons, and outward propagation. I predicted and showed the neuropathological correlate of this, namely loss of motor neurons as a function of distance from site of onset. I generated a hypothesis to model neuroanatomical propagation and showed applicability to familial ALS and to frontotemporal dementia (FTD), a related neurodegeneration. I have emphasized the role of contiguous or local spread, which is a distinct pattern of propagation from network spread, a theme also emerging in the literature. These ideas are helping formulate ideas about phenotypes, progression, neuropathology, mechanisms of cell-to-cell spread, regional therapies with stem cells, and biomarker identification.

1. Ravits J, et al. Neurology. 2007 May 8;68(19):1571-5. PubMed PMID: 17485643. 2. Ravits J, et al. Neurology. 2007 May 8;68(19):1576-82. PubMed PMID: 17485644. 3. Ravits JM and La Spada AR. Neurology. 2009 Sep 8;73(10):805-11. PubMed PMID:

19738176. 4. Ravits J. Exp Neurol. 2014 Dec;262 Pt B:121-6. PubMed PMID: 25108067.

2. Genomic profiling: I exploited the propagating nature of ALS to identify locations of residual (undegenerated) motor neurons that did not but were at risk to succumb to the process of neurodegeneration based on anatomic location. I created a repository of human nervous systems with short post mortem intervals, laser captured residual motor neurons, profiled their transcriptome, and used innovative unbiased bioinformatics to identify novel pathways, especially enriched in extracellular matrix, transmembrane signaling and downregulation of microRNAs. Importantly, these identifications were made by an unbiased or discovery approach, and are distinctly different from pathways, such as axonal transport, apoptosis, RNA processing, protein clearance, or mitochondrial pathways. How far upstream or downstream these pathways are remains to be established.

1. Rabin SJ, et al. Hum Mol Genet. 2010 Jan 15;19(2):313-28. PubMed PMID: 19864493. 2. Emde A,et al. EMBO J. 2015 Nov 3;34(21):2633-51. PubMed PMID: 26330466.


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3. ALS Neuropathology: Consistent with my interest in neuroanatomical propagation of ALS, I am studying the post-mortem ALS CNS neuropathologically. I have created a tissue repositories from patients whose phenotypes have been well-characterized and am studying the anatomy of key neuropathological features. In this, I am seeking to characterize the distribution of changes between brain and spinal cord, comparing neuronal pathology in Betz cells of layer 5b of motor cortex and in alpha motor neurons in lamina IX of anterior horns, and the spread of pathology based on somatotopic anatomy within these regions. Related to this, I am studying mechanisms of neuronal death, such as by the RIPK1-mediated necroptosis. RIPK1 is a key mediator of the innate immune response that regulates both inflammation and cell death.

1. Bodansky A et al. Amyotroph Lateral Scler. 2010 May 3;11(3):321-7. PubMed PMID: 20225928.

2. Song F, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Mar;15(1-2):77-83. PubMed PMID: 24229388.

3. Ito Y, et al. Science. 2016 Aug 5;353(6299):603-8. PubMed PMID: 27493188.

4. Braak H et al. Acta Neuropathologica. 2017 Jan;133(1):79-90. PMID:27757524 (in process).

5. Da Cruz S et al. Acta Neuropathological. 2017. Feb PMID:28247063 (in process). 4. CORF72 ALS:

I am working on identifying mechanisms and therapeutic targets of neuronal degenerations in ALS caused by repeat expansion mutations in C9orf72. There is strong evidence for gain-of-function either at the RNA or protein levels from both the sense and antisense directions. Our work (and that of others in the field) has shown a significant presence of transcripts from the antisense strand as well as the sense strand, essentially doubling the number of candidates for therapeutic targeting. My current research is trying to compare the relative contributions of these oppositely directed transcripts to pathogenesis in a mouse model and in humans neuropathology and trying to ascertain the most appropriate target for antisense oligonucleotide (ASO) therapies. ASOs are exquisitely engineered gene therapies and have the potential to create a true revolution in neurotherapeutics. C9forf72-ALS/FTD, which is as much as 8% of ALS and FTD patients, will be a critical testing ground. Related to this, my translational research group is participating in the international effort to develop ASOs for ALS by intrathecal delivery, beginning with mutSOD1-associated ALS.

1. *Lagier-Tourenne C, *Baughn M, et al. Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9. PubMed PMID: 24170860.

2. Sareen D, et al. Sci Transl Med. 2013 Oct 23;5(208):208ra149. PubMed PMID: 24154603.

3. Jiang J, et al Neuron. 2016 May 4;90(3):535-50. PubMed PMID: 27112497. TRANSLATIONAL RESEARCH ACTIVITIES I: CLINICAL TRIALS AND TRIALS OVERSIGHT

Principal investigator, Seattle site, Double-blind Trial of Human Recombinant Ciliary Neurotrophic Factor in ALS, 1993-5. Study included three sub-studies: 1) Natural History, 2) Clinical Testing, & 3) Post Testing. Funded by Synergen Corporation.

Principal investigator, Seattle site of Western ALS (WALS) Study Group, Double-blind trial of Gabapentin in ALS, 1995-6. Funded by WALS (with limited corporate support).


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Principal investigator, Early access program of Riluzole, 1995-6. Funded by Rhone-Poulenc Rorer, Inc.

Principal investigator, Early access program for Myotrophin in ALS, 1996-9. Funded by Cephalon, Inc.

Principal investigator, Seattle site of Western ALS Study Group: Follow-up Study of Double-blind trial of Gabapentin in ALS, 1996-9. Investigator initiated study funded by WALS Study Group (with limited corporate support).

Principle investigator, Seattle site of Western ALS Study Group, Double-blind trial of minocycline in ALS, 2002-2004. Funded by NIH RO1 NS045294-01.

Principle Investigator, University of California, San Diego site, Phase IIb study to assess the effect of CK-2017357, 2012-13. Funded by Cytokinetics, Inc.

Member, Steering Committee of Muscular Dystrophy Association’s and ALS Association’s Safety and Efficacy Trial of Synapse’s NeuRx® Diaphragm Pacing System (DPS) in ALS patients, 2012 to 2014. No funding for this role.

Member and Chairman, Data and Safety Monitoring Board, A Safety and Tolerability Study of Mexiletine in Patients with Sporadic Amyotrophic Lateral Sclerosis (SALS) MX-ALS-001, 2013-2014. No funding for this role.

Member, Clinical Adjudication Committee, Alnylam Pharmaceuticals, Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy/FAP), Alnylam ALN-TTR02-004 Study (APOLLO). 2013-present. Funded by Alnylam Pharmaceuticals, Inc..

Principle Investigator, University of California San Diego site, VITALITY ALS: A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Triasemtiv in Patients with Amyotrophic Lateral Sclerosis, 2015-present. Funded by Cytokinetics Inc, South San Francisco, CA

Principle Investigator, University of California, San Diego site, A Phase 1 Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerossis, 2016-present. Funded by Biogen MA Inc, Cambridge MA.

TRANSLATIONAL RESEARCH ACTIVITIES II: PHENOTYPING AND BIOSAMPLES

Principle Investigator, CNS and blood biorepositories, Virginia Mason Medical Center, Investigator initiated effort 2002-2011 (transferred to University of California, San Diego). Self-funded.

Principle Investigator, University of California, San Diego site, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe), Initiative of the National Center for Advancing Translational Sciences (NCATS), U54 NIH Rare Disease Consortium (U54NS092091), Benatar PI, 8/1/2015-7/31/2020.

Principle Investigator, University of California, San Diego site, C9 Natural History, 2015-present. Sponsored by Biogen and ALSA.

Principle Investigator, University of California, San Diego site, Biogen 999AS003 ALS Methodology Study of Novel Electrophysiological, Physical, and Imaging Outcome Measures to Assess the Progression of Amyotrophic Lateral Sclerosis, 2016-present. Funded by Biogen MA Inc, Cambridge MA.

Principle Investigator, University of California, San Diego site, Target ALS Principle Investigator, University of California, San Diego site, ALSA Proposal for Adding Biofluids

to Target ALS Postmortem Tissue Core BASIC RESEARCH ACTIVITIES

Principle investigator, ALS Tissue Bank and Neuro-genomics Lab, Benaroya Research Institute, (2001-present).

Grant from Virginia Mason Research Center, $39,000. (2001) Grant from Juniper Foundation, Seattle, WA, 2003-5, $30,000 (2002-2004)


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Grant from Moyer Foundation, Seattle, WA, 2003, $85,000 (Oct, 2003) Grant from Moyer Foundation, Seattle, WA, 2004, $85,000 (Dec, 2004) Philanthropic donations, various private donors and foundations, ~$100,000-$200,000 per

year (2005-present) Principle investigator, Microgenomic Studies of ALS, NIH R21 NS051738-01A1, $275,000 Jan 1,

2006-Dec 31, 2007 Principle investigator, Pilot 3, Neuronal and Matrix Interactions and Motor neuron injury and repair,

BRI Center for Inflammation and Tissue Repair, USAMRMC Proposal #06054001, Jan 2006-present.

Principle investigator, Microsoft Research Research Program Development, Microsoft Corp, Redmond, WA, ALS Genomics, Apr 1, 2008-Mar 31, 2010, $1,000,000 (renewable Apr 1, 2010-2011, $500,000). 1 year extension 12/31/10-12/30/11, $200,000

Sub Investigator, RNA Dysregulation in ALS, NIH 1RC2NS069473 (PI: Darnell), $157,930, 9/30/09-9/29/11

Principle investigator, investigating transcriptome changes in hexanucleotide repeat expanded C9orf72 ALS, ALS Association Grant 5356S3, $240,000 August 1, 2012 to July 31, 2015

Co-Investigatory, Pilot Project on Transcriptome Profiling of ALS, Project ALS and Packard Foundation (“P2ALS”), Co-PI with Don Cleveland, $104,000

Sub-Investigator, CIRM RB4-06045: Stem cell models to analyze the role of mutated C9ORF72 in neurodegeneration, CIRM RFA 11-03 Basic Biology IV Awards, Gene Yeo PI, $1,393,200. 03/01/13-02/29/16

Project Investigator, Target ALS, C9orf72 ALS: Testing Targets and Developing Models, Project 4: C9orf72 ALS/FTD Treatment with Antisense Oligonucleotides (ASOs), $390,000. 05/01/2013-04/30/2016.

Principle investigator, Developing ASO Therapy for Repeat Expanded C9orf72 ALS-FTD, NIH R01NS088578, $2,142,940 July 1, 2014-Dec 31, 2019

NATIONAL/INTERNATIONAL PRESENTATIONS

1. No morphometric abnormality of the recurrent laryngeal nerve in spastic dysphonia. 8th International Congress of Neuropathy, Washington, DC, September 1978.

2. Primary writing tremor and primary writing myoclonus: Unusual task-specific movement disorders. Boston Society of Psychiatry and Neurology, Boston, MA, February 1984.

3. Primary writing tremor and myoclonic writer's cramp. 31st Annual Meeting of the American Association of Electromyography and Electrodiagnosis, Kansas City, MO, September 1984.

4. Spontaneous reticular myoclonus. 32nd Annual Meeting of the American Association of Electromyography and Electrodiagnosis, Las Vegas, NV, October 1985.

5. Adult-onset body tic. 38th Annual Meeting of the American Academy of Neurology, New Orleans, LA, May 1986.

6. A comparative study of electrophysiologic tests of autonomic function in patients with primary autonomic failure. 33rd Annual Meeting of the American Association of Electromyography and Electrodiagnosis, Boston, MA, September 1986.

7. Autonomic function testing workshops. 34th Annual Meeting of the American Association of Electromyography and Electrodiagnosis, San Antonio, TX, December 1987; 35th annual meeting, San Diego, CA, September 1988; 36th annual meeting, Washington, DC, September 1989; 37th annual meeting, Chicago, IL, 1990; 38th annual meeting, Vancouver, BC, Canada, 1991; 39th annual meeting, Charleston, SC, 1992; 42nd annual meeting, Montreal, Canada, 1995, 43rd annual meeting, Minneapolis, MN, 1996 and 45th annual meeting, Orlando, FL, 1998; 46th annual meeting, Vancoever,Canada.1999; 49th annual meeting, Albuquerque, NM, 2001; 50th annual meeting, Toronto, Canada, 2002.

8. Clinical and electrophysiologic findings in Fabry's disease. 37th Annual Meeting of the American Association of Electrodiagnostic Medicine, Chicago, IL, September 1990.


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9. Testing cardiac and vascular autonomic reflexes. 41st annual meeting of American Association of Electrodiagnostic Medicine, Course on Autonomic Function, San Francisco, CA, 1994.

10. Update on Autonomic Function. Annual meeting of the American College of Occupational and Environmental Medicine, course on Advances and Controversies in Clinical Medicine. October 1995.

11. Autonomic Neuropathy, Breakfast Seminar. 43rd Annual Meeting of the Annual Meeting of the American Association of Electromyography and Electrodiagnosis, AAEM, Mpls, MN, 1996 and 45th Annual Meeting, Orlando, FL, 1998.

12. Demyelinating Neuropathies. Morning Seminar given at 60th Annual Meeting of the American Academy of Physical Medicine and Rehabilitation, November 6, 1998.

13. Assessment of Autonomic Function. 24th Annual Electrodiagnosis and Clinical Neurophysiology: A High Intensity Review, Rehabilitation Institute of Chicago, Chicago, IL, March 15, 2001.

14. Quantitative EMG. 24th Annual Electrodiagnosis and Clinical Neurophysiology: A High Intensity Review, Rehabilitation Institute of Chicago, Chicago, IL, March 15, 2001.

15. Dystonia: Workshop for the Use of Botulinum Toxins, Co-sponsored by the Movement Disorder Society, the National Institutes of Health and the Foundation for the Advanced Education of the Sciences (NIH/FAES), Seattle, WA, October 12, 2001.

16. Advanced Treatment of Dystonia and Spasticity: Workshop Demonstrating the Use of Botulinum Toxin. Program of the American Academy of Neurology, The Movement Disorder Society and WE MOVE, Seattle/Bellevue; October 16, 2004.

17. Motor Neuron Isolation by Tissue Microdissection for Biochemistry in ALS Nervous Systems, poster presentation at the 15th International Symposium on ALS/MND, Philadelphia, December 2, 2004.

18. RNA Assessment in ALS Nervous Systems and Motor Neurons Isolated by Microdissection in ALS Nervous Systems, poster presentation at the 15th International Symposium on ALS/MND, Philadelphia, December 2, 2004.

19. Focality of ALS: 3-D Topography of Lower Motor Neuron Degeneration in Post-Mortem Nervous Systems and the Neuropathologic Gradient of Degeneration. Poster presentation at the 58th Meeting of the American Academy of Neurology, San Diego, CA, April 5, 2006.

20. ALS at the Onset: Topography of UMN and LMN Loss; platform presentation at the 17th International Symposium on ALS/MND, Yokohama, Japan, December 2, 2006.

21. Differential Genes Set Expression in SALS Motor Neuron and Anterior Horn Enriched RNA Pools Using Whole Genome Oligonucleotide Microarray. 18th International Symposium on ALS/MND. Toronto, Canada, Dec 1, 2007.

22. Correlation Between Genome-wide SNP and Whole Genome Expression: Discovery-based Pursuit of SALS Pathobiology. 18th International Symposium on ALS/MND. Toronto, Canada, Dec 1, 2007.

23. Deciphering ALS. Canadian ALS Research Annual Meeting, Toronto, Canada, May, 2008. 24. Differential Genes Set Expression in G93A Transgenic Mice Using Whole Genome

Oligonucleotide Microarray. 19th International Symposium on ALS/MND. Birmingham, UK, Nov 3, 2008.

25. Distribution of TDP-43 Intracytoplasmic Inclusions in the ALS Gradient of Neurodegeneration. 19th International Symposium on ALS/MND. Birmingham, UK, Nov 3, 2008.

26. ALS Focality: Motor Neuron Degeneration in the House of Mirrors. 15th Annual Halseth ALS Lecture, University of Colorado Health Sciences University, Aurora/Denver, CO, February 11, 2009.

27. Linkage between disease onset and progression, 3rd International Research Workshop on Frontotemporal Dementia in ALS, London, Ontario CA, June 22, 2009.


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28. ALS Microgeneomics. Universität Ulm, Ulm, Germany, Dec. 7, 2009. 29. The Growing Evidence of Abnormal RNA Processing in ALS. Wayne State University

Department of Molecular Medicine, Detroit, MI, Jan. 7, 2010. 30. ALS Focality, Looking for Motor Neuron Degeneration in the House of Mirrors. Wayne State

University Department of Neurology Grand Rounds, Detroit, MI, Jan. 8, 2010. 31. Deciphering ALS: Motor Neuron Degeneration in a House of Mirrors. University of California,

San Diego, Neurology Grand Rounds, San Diego, CA, July 9, 2010 32. ALS Motor Phenotype Heterogeneity, Focality and Spread. Cleveland Clinic Neurology

Grand Rounds, Cleveland, OH, Aug. 9, 2010. 33. ALS Focality and Spread: Following the Trail, Reading the Cairns. Cedars Sinai Medical

Center Neurology Grand Rounds, Los Angeles, CA, Oct. 22, 2010. 34. ALS phenotype and ALS pathogenesis, NINDS-Columbia University invited conference,

Sept. 7-9, 2011, Tarrytown House, Tarrytown (Westchester), NY. 35. Bounded in a Nutshell and Counting an Infinite Space: Profiling the Transcriptome in SALS

Motor Neurons. CALS Research Symposium, Los Angeles, CA Sept. 30, 2011 36. What Heterogeneity of ALS Phenotype May Be Telling Us About Pathobiology. Plenary

Address, 22nd International Symposium on ALS/MND. Sydney, AU, Nov 30, 2011. 37. Regional Spread in ALS: Mechanisms and Pathogenesis. 2nd Annual Neuromuscular

Colloquium, Newport Beach, CA, May 4, 2012. 38. Regional Spread in ALS: Mechanisms and Pathogenesis, World Federation of Neurology

Outreach Courses in ALS and FTD, Beijing China, June 22, 2012 39. Regional Spread in ALS: Mechanisms and Pathogenesis, World Federation of Neurology

Outreach Courses in ALS and FTD, Ulaanbaatar, Mongolia, June 25, 2012 40. Finding ALS: Transcriptome dysregulation in ALS motor neurons. Project ALS and Columbia

University Motor Neuron Disease Center, New York, NY, September 28, 2012. 41. Chairman, Special Interest Group on Neuromuscular Diseases, 2012 Annual Meeting of the

American Neurological Association, Boston, MA, Oct. 8, 2012. 42. Repeat Expanded C9ORF72 ALS-FTD: Expanding Phenotypes and Spreading Disease.

Research Symposium of the Annual Society for Neurosciences, New Orleans, LA, October 15, 2012.

43. Invited Speaker, Annual ALS-Patient Symposium, 15th November 15, 2012, Vancouver, Canada.

44. Spatial localization in ALS: Update and expanding the model; California ALS Summit Meeting, Los Angeles, Nov 16, 2012.

45. Identifying mechanisms and therapy for C9orf72 ALS/FTD; California ALS Summit Meeting, Los Angeles, CA, Nov 17, 2012.

46. C9ORF72 ALS/FTD: Expanding phenotypes and focusing therapy. Neurodegeneration Seminar Series, Shiley-Marcos Alzheimer’s Disease Research Consortium, UCSD, San Diego, Dec 18, 2012

47. What is C9ORF72-ALS/FTD and why is it transforming the way we are thinking about neurodegeneration, UCSD Neuroscience Grand Rounds, Feb 8, 2013

48. Expanding Neurodegenerations: C90RF72 Amyotrophic Lateral Sclerosis/ Frontotemporal Dementia, Southern California Alzheimer’s Disease Centers 2013 Research Symposium, University of California, Irvine, April 5, 2013.

49. Clinical-Pathological Observations on Spread in ALS: Propagation Biology. Banbury Conference on the Development and Evolution of the Human Motor System in relation to ALS & FTD, Cold Springs Harbor Research Laboratories, Long Island, NY, April 16, 2013.

50. Program Director, Symposium on Cell Therapy for ALS—Testing the limits: What should we use as preclinical standards for initiation of clinical trials? Stanford Consortium for Regenerative Medicine, La Jolla, CA, May 6, 2013.


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51. Mechanisms of Propagation in ALS, Hot Topics in Neuroscience, 54th Annual Meeting of Japanese Neurological Society, May 30, 2013.

52. Chairman, Special Interest Group on Neuromuscular Diseases, Annual Meeting of the American Neurological Association, New Orleans, LA, Oct 15, 2013.

53. Expanding neurodegenerations: Update on C9orf72, American Neurological Association, New Orleans, LA, Oct 15, 2013.

54. More evidence supporting perturbation in extracellular and transmembrane domains and of protein signaling by transcriptome analysis of motor neurons from sporadic ALS spinal cords. 24th International Symposium on ALS/MND, Milan, Italy, Dec 10, 2013.

55. Focality, stochasticism, and spread in ALS and comparisons and contrasts to FTD. Center for Memory and Aging, University of California, San Francisco, San Francisco, CA, Mar 15, 2014.

56. ALS and neuroanatomic propagation of motor neuron degeneration. Bienniel International Motoneuron Meeting, Dalhousie University, Halifax, Nova Scotia, Canada, June 16, 2014

57. C9orf72 Recent developments and the path to the clinic. ALS Association Greater Philadelphia Chapter, Philadelphia, PA, Oct 19, 2014.

58. Focality, stochasticity and neuro-anatomic propagation in ALS pathogenesis. ALS Association Investigator Research Workshop. Philadelphia, PA, Oct 20, 2014.

59. Focality, stochasticity and neuro-anatommic propagation in ALS pathogenesis. The Stephen F. Heinemann Seminar in Neuroscience. Sanford-Burnham Medical Research Institute. La Jolla, CA, Oct 31, 2014

60. Highlights of 2014: Clinical and Care Management Research. 25th International Symposium on ALS/MND. Brussels, Belgium. Dec 7, 2014

61. What is C9orf72 and why is it changing the way we are thinking about neurodegenerations? T Evans Wyckoff in Neuroscience, Virginia Mason Medical Center, Seattle, WA, Oct 2, 2015.

62. Update on C9orf72 ALS and FTD. University of Ulm, Ulm, Germany, Oct 12, 2015. 63. Focality and neuroanatomic propagation in ALS: Deconstructing clinical and

neuropathological phenotypes, Institute of Molecular Life Sciences, University of Zurich, University of Zurich, Zurich Switzerland, Oct 30, 2015.

64. Understanding ALS Disease Progression, Plenary Talk for the Clinical-Pathological Correlates of Disease Progression Session at the 26th International Symposium on ALS/MND, Orlando, CA, Dec 12, 2015.

65. ALS: Past, present and future. Keynote Speaker, Evergreen State ALS Association, Seattle, WA, April 1, 2016

66. What is C9orf72 ALS and why is it changing the way we think about neurodegenerations? Medical College of Wisconsin Grand Rounds, Milwaukee, WI, April 29, 2016.

67. ALS in the canary in the tunnel of healthcare. Keynote Speaker, Wisconsin Annual ALS Symposium, Milwaukee, WI, April 30, 2016.

68. What is repeat expanded C9orf72 ALS and FTD, and why is it changing the way we are thinking about neurodegeneration? Annual Litchfield Lectures, Sherrington Library, John Radcliffe Hospital and University of Oxford, Oxford, England, Aug 4, 2016.

69. Neuroanatomical propagation in ALS neurodegeneration: Where did this start and where is it going? Annual Litchfield Lectures, Nuffield Department of Clinical Neurosciences, John Radcliffe Hospital and University of Oxford, Oxford, England, Aug 5, 2016.

70. ALS Genotypes and phenotypes: Complex genetics and complex neuroanatomy. Zing Conference on Neuroanatomic Propagation in Neurodegenerative Diseases, Malahide (Dublin), Ireland, Aug. 11, 2016.

71. ALS Research Update: Meeting of the National ALS Association Board of Trustees, Irvine, CA, Feb 2, 2017.


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72. Does ALS begin as a point source? Address delivered upon receiving the Sheila Essay Award for ALS Reserch, American Academy of Neurology, Boston, MA, Apr 23, 2017.

73. Profiling microRNAs in ALS. Annual Meeting of Target ALS, Boston, MA, May 15, 2017 FIVE MOST IMPORTANT PUBLICATIONS

1. Ravits J, Paul P, Jorg C: Focality of upper and lower motor neuron degeneration at the clinical onset of ALS. Neurology 2007;68:1571-1575.

2. Ravits J, Laurie P, Fan Y, and Moore DH: Implications of ALS focality: rostral-caudal distribution of lower motor neuron loss postmortem. Neurology 2007;68:1576-1582.

3. Ravits J, LaSpada A: Motor phenotype heterogeneity, focality, and spread in ALS: deconstructing motor neuron degeneration. Neurology 2009; 73: 805– 811.

4. Rabin S, Kim H, Baughn M, Libby R, Kim Y, Fan Y, La Spada A, Libby RT, Stone B, Ravits J: Sporadic ALS has compartment-specific aberrant exon splicing and perturbation of cell-matrix adhesion biology. Human Molecular Genetics 2010;19:313–328.

5. Lagier-Tourenne*, C, Baughn*, M, Rigo, F, Sun, S, Liu, P, Li, H-R, Jiang, Jie, Watt, A, Chun,S, Katz, M, Qiu, J, Sun, Y, Ling, S-C, Shu, Q, Polymenidou, M, Drenner, K, Arates, JW, McAlonis Mm, Markmiller S, Hutt, KR, Pizzo, DP, Cady, J, Harms, MB, Baloh, RH, VandenBerg, SR, Yeo, GW, Fun X-D, Bennet CF, Cleveland, DW*, and Ravits, J* (*=shared contributions): Targeted degradation of sense and antisense C9orf72 nuclear RNA foci as therapy for amyotrophic lateral sclerosis and frontotemporal degeneration. Proc Natl Acad Sci U S A. 2013 Nov 19;110 (47):E4530-9. PMID: 24170860

PUBLICATIONS

6. Ravits JM, Aronson AE, DeSanto LW, Dyck PJ: No morphometric abnormality of the recurrent laryngeal nerve in spastic dysphonia. Neurology 1979; 29:1376-1382.

7. Ravits JM, Seybold ME: Amaurosis fugax from angle-closure glaucoma. Arch Neuro 1984; 41:991-993.

8. Ravits J, Hallett M, Baker M, Wilkins D: Primary writing tremor and myoclonic writer's cramp. Neurology 1985; 35:1387-1391.

9. Ravits J, Hallett M: Pathophysiology of hemifacial spasm (letter). Neurology 1986; 36:591-3.

10. Dalakas M, Elder G, Hallett M, Ravits J, et al: A long-term follow-up study of patients with post-poliomyelitis neuromuscular symptoms. N Engl J Med 1986; 314:959-963.

11. Ravits J: Autonomic Function Testing (An AAEE workshop). American Association of EMG and Electrodiagnosis. Rochester, MN, October 1987, pp. 1-4. (Revised 1990)

12. Ravits J: Myasthenia gravis, a well-understood neuromuscular disorder. Postgrad Med 1988; 83:219-222.

13. Nilsson J, Ravits J, Hallett M: Stimulus artifact compensation using biphasic stimulation. Muscle Nerve 1988; 11:597-602.

14. Geller B, Hallett M, Ravits J: Botulinum therapy in hemifacial spasm: clinical and electrophysiologic studies. Muscle Nerve 1989; 12:716-722.

15. Ravits J, Hallett M, Baker M, Nilsson J, Dalakas M: Clinical and electromyographic studies of postpoliomyelitis muscular atrophy. Muscle Nerve 1990; 13:667-674.

16. Hallett M, Ravits J, Dubinsky R, et al: A double-blind trial of isoniazid for essential tremor and other action tremors. Movement Disorders, 1991; 6(3):253-256.

17. Demirer T, Ravits J, Aboulafia D: Myasthenic (Eaton-Lambert) syndrome associated with pulmonary large-cell neuroendocrine carcinoma. S Med J, 1994; 87(11):1186-9.

18. Ravits J, Hallett M, Nilsson J, et al: Electrophysiological studies in idiopathic autonomic failure syndromes. Muscle Nerve, 1996; 19:758-763.

19. Miller RG et al and the ALS CNTF Treatment Study (ACTS) Group (including Ravits J): A, placebo-controlled trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Ann Neurol, 1996,39(2) 256-60.


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20. McGuire D et al and the SSNJV/CNTF ALS Study Group (including Ravits J): Relationship of the Tufts Quantitative Neuromuscular Exam (TQNE) and the Sickness Impact Profile (SIP) in measuring progression of ALS. Neurology, 1996; 46(5):1442-4.

21. Miller RG, et al (including Ravits J): Placebo-controlled trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology, 1996; 47:1383-1388.

22. Ravits J: AAEM Minimonograph #48: Autonomic Nervous System Testing. Muscle Nerve, 1997;20:919-937.

23. McGuire D et al and the Syntex-Synergen ALS/CNTF Study Group (including Ravits J): A brief quality-of-life measure for ALS clinical trials based on subset of items from the sickness impact profile. J Neurol Sci. 1997;152 Suppl 1:S18-22.

24. Hoagland RJ et al and The Syntex/Synergen Neuroscience Joint Venture rhCNTF ALS Study Group (including Ravits J): Reliability of maximal voluntary isometric contraction testing in a multicenter study of patients with amyotrophic lateral sclerosis. Muscle Nerve 1997;20 (6):691-5.

25. Ross MA, Miller RG Berchert L et al and the rhCNTF ALS Study Group (including Ravits J): Toward earlier diagnosis of amyotrophic lateral sclerosis: Revised criteria. Neurology, 1998;50:768-772.

26. Ravits J: Neurology epitome: update on treatment of peripheral neuropathy. West J Med 168:268-9, 1998.

27. Fernandez M, McClain ME, Martinez RA, Snow K, Lipe H, Ravits J, Bird TD, & LaSpada AR: Late-onset SCA2: 33 CAG repeats are sufficient to cause disease. Neurology 2000;55(4):569-572.

28. Miller RG, Moore D, Gelinas DF et al (including Ravits J): Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001;56:843-848.

29. Bryan WW, Hoagland RJ, and The Syntex/Synergen Neuroscience Joint Venture rhCNTF ALS Study Group (including Ravits J): Can we eliminate placebo in ALS clinical trials? ALS and other Motor Neuron Diseases 2003;4:11-15.

30. Andersen PM, Sims KB, Xin WW, Kiely R, O'Neill G, Ravits J, Pioro E, Harati Y, Brower RD, Levine JS, Heinicke HU, Seltzer W, Boss M, Brown RH. Sixteen novel mutations in the gene encoding CuZn-superoxide dismutase in ALS. Amyotrophic Lateral Sclerosis and other motor neuron disorders, 2003; 2: 62-73.

31. Ravits J: Sporadic amyotrophic lateral sclerosis—a hypothesis of persisting enteroviral infection. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2005;6:77-87.

32. Weiss MD, Ravits JM, Schuman N, Carter GT: A4V superoxide dismutase mutation in apparently sporadic ALS. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2006 Mar;7(1):61-3.

33. Mackenzie IRA, Bigio EH, Ince PG, Geser F, Neumann M, Cairns NJ, Kwong LW, Forman MS, Ravits J, Shaw PJ, Stewart H, McClusky L, Kretzschmar HA, Monoranu C, Highley R, Kirby J, Siddique T, Lee V M-Y, Trojanowski JQ: Pathological TDP-43 distinguishes sporadic ALS from ALS with SOD-1 mutations. Ann Neurol 2007;61:427-434.

34. Ravits J, Paul P, Jorg C: Focality of upper and lower motor neuron degeneration at the clinical onset of ALS. Neurology 2007;68:1571-1575.

35. Ravits J, Laurie P, Fan Y, and Moore DH: Implications of ALS focality: rostral-caudal distribution of lower motor neuron loss postmortem. Neurology 2007;68:1576-1582.

36. Gordon PH, Moore DH, Miller RG et al (incl Ravits J): Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomized trial. Lancet Neurology 2007;6:1045-53.

37. Xiao S, Tjostheim S, Horne P, Fan Y, Ravits J, Strong M, Fan Y, Robertson J: An aggregate-inducing peripherin isoform generated through intron retention is upregulated in amyotrophic lateral sclerosis and associated with disease pathology. J Neurosci. 2008;28(8):1833-40.


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38. Kim YJ, Fan Y, Laurie P, 'Hugo' Kim JM, Ravits J: No evidence of HIV pol gene in spinal cord tissues in sporadic ALS by real-time RT-PCR. Amyotroph Lateral Scler. 2009;Mar 25:1-9.

39. Ravits J, LaSpada A: Motor phenotype heterogeneity, focality, and spread in ALS: deconstructing motor neuron degeneration. Neurology 2009; 73: 805– 811.

40. Rabin S, Kim H, Baughn M, Libby R, Kim Y, Fan Y, La Spada A, Libby RT, Stone B, Ravits J: Sporadic ALS has compartment-specific aberrant exon splicing and perturbation of cell-matrix adhesion biology. Human Molecular Genetics 2010;19:313–328.

41. Bodansky A, Kim H, Tempest L, Velagupudi A, Libby RT, Ravits J: TDP-43 and ubiquitinated cytoplasmic aggregates in ALS are low frequency and widely distributed in the lower motor neuron columns independent of disease spread. Amyotroph Lateral Scler. 2010 May 3;11(3):321-7.

42. Song F, Chiang P, Wang J, Ravits J and Loeb JA: Aberrant neuregulin1 signaling in amyotrophic lateral sclerosis. J Neuropathol Exp Neurol. 2012 Feb;71(2):104-15.

43. Lagier-Tourenne C, Polymenidou M, Hutt KR, Vu AQ, Baughn M, Huelga SC, Clutario KM, Ling SC, Liang TY, Mazur C, Wancewicz E, Kim AS, Watt A, Freier S, Hicks GG, Donohue JP, Shiue L, Bennett CF, Ravits J, Cleveland DW, Yeo GW. Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs. Nat Neurosci. 2012 Nov;15(11):1488-97.

44. Turner MR, Hardiman O, Benatar M, Brooks BR, Chio A, de Carvalho M, Ince PG, Lin C, Miller RG, Mitsumoto H, Nicholson G, Ravits J, Shaw PJ, Swash M, Talbot K, Traynor BJ, Van den Berg LH, Veldink JH, Vucic S, Kiernan MC. Controversies and priorities in amyotrophic lateral sclerosis. Lancet Neurol. 2013 Mar;12(3):310-22.

45. Benatar M, Wuu J, and Ravits J: Opportunity and Innovation in Studying Pre-Symptomatic ALS. Muscle Nerve. 2013 May;47(5):629-31 PMID: 23494825

46. Harms, MB, Cady, J, Zaidman, C, Cooper, P, Bali, T Allred, P, Cruchaga, C Baugh M,, Libby RT, Pestronk, A, Goate, A, Ravits, J, Baloh, RH: Lack of C9ORF72 coding mutations supports a gain of function for repeat expansions in ALS. Neurobiol Aging. 2013 Apr 16. doi:pii: S0197-4580(13)00106-1 PMID: 23597494

47. Ravits J et al: De-cyphering ALS: What phenotype, neuropathology and genetics are telling us about pathobiology. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14 Suppl 1:5-18. PMID: 23678876.

48. Sareen D, O'Rourke JG, Meera P, Muhammad AK, Grant S, Simpkinson M, Bell S, Carmona S, Ornelas L, Sahabian A, Gendron T, Petrucelli L, Baughn M, Ravits J, Harms MB, Rigo F, Bennett CF, Otis TS, Svendsen CN, Baloh RH: Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion. Sci Transl Med. 2013 Oct 23;5(208):208ra149. PMID: 24154603.

49. Lagier-Tourenne*, C, Baughn*, M, Rigo, F, Sun, S, Liu, P, Li, H-R, Jiang, Jie, Watt, A, Chun,S, Katz, M, Qiu, J, Sun, Y, Ling, S-C, Shu, Q, Polymenidou, M, Drenner, K, Arates, JW, McAlonis Mm, Markmiller S, Hutt, KR, Pizzo, DP, Cady, J, Harms, MB, Baloh, RH, VandenBerg, SR, Yeo, GW, Fun X-D, Bennet CF, Cleveland+, DW, and Ravits, J+: Targeted degradation of sense and antisense C9orf72 nuclear RNA foci as therapy for amyotrophic lateral sclerosis and frontotemporal degeneration. Proc Natl Acad Sci U S A. 2013 Nov 19;110 (47):E4530-9. PMID: 24170860

50. Song F, Chiang P, Ravits J, Loeb JA. Activation of microglial neuregulin1 signaling in the corticospinal tracts of ALS patients with upper motor neuron signs. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Nov 14. PMID:24229388.

51. Meyer, K, Ferraiuolo, L, Miranda CJ, Likhite, S, McElroy, S, Renusch, S, Ditsworth, D, Lagier-Tourenne, C, Smith, RA, Ravits, J, Burghes A, Shaw, PJ, Cleveland, DW, Stephen J. Kolb, SJ, Kaspar BK Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS. Proc Natl Acad Sci U S A. 2014 (cgi/doi/10.1073/pnas.1314085111)


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52. Bruijn L, Cudkowicz M and the ALS Clinic Trials Working Group (incl Ravits, J): Opportunities for improving therapy development in ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2014, 2013; Early Online: 1–5. 2014; 15: 169–173

53. Cady, J, Koval, E, Benitez, B, Zaidman, C, Jockel-Balsarotti, J, Allred, P, Baloh, B, Ravits, J, Simpson, E, Appel, S, Pestronk, A, Goate A, Miller, T, Cruchaga,C, Harms, M: The TREM2 variant p.R47H is a risk factor for sporadic amyotrophic lateral sclerosis. JAMA Neurology (2014 Feb 17. doi: 10.1001/jamaneurol.2013.6237.) PMID: 24535663

54. Ravits J: Focality, stochasticity and neuroanatomic propagation in ALS pathogenesis. Experimental Neurology, 2014 Aug 6. pii: S0014-4886(14)00249-0. doi: 10.1016/j.expneurol.2014.07.021. PMID: 25108067

55. Gallardo, G, Barowski, J, Ravits, J, Siddique, T, Lingrel, JB, Robertson J, Steen, H, Bonni, A: An α2-Na/K ATPase/α-adducin complex in astrocytes triggers non-cell autonomous neurodegeneration. Cell. Nat Neurosci. 2014 Oct 26. doi: 10.1038/nn.3853. [Epub ahead of print]

56. Cady, Janet; Allred, Peggy; Bali, Taha; Pestronk, Alan; Goate, Alison; Miller, Timothy; Mitra, Robi; Ravits, John; Harms, Matthew; Baloh, Robert. ALS onset is influenced by the burden of rare variants in known ALS genes. Annals Neurology. 2014 doi:10.1002/ana.24306

57. Cirulli, ET, Lasseigne, BN, Petrovski, S, Sapp P , Dion P, Leblond C, Couthouis, J, Lu Y-F, Wang Q, Keebler, J, Krueger, BJ, Ren, N, Levy SE, Boone, BE, Wimbish, JR, Jones, LW, Jones, A, Carulli, J, Day-Williams, A, Staropoli, J, Xin, WW, Trojanowski, JQ, Elman, L, McCluskey, L, Grossman, M, Shneider, N, Chung,W, Ravits, JM,. Glass, JR, Sims, KB, Van Deerlin, V, Maniatis, T, Harper,W, Allen, A, Bedlack, RS, Gitler, A, Rouleau, G, Brown, R, Harms, MB, Cooper, G, Harris, T, Myers, RM, Goldstein, DB: The exomes of 3,500 patients identify new ALS genes and pathways. Science. 2015 Mar 27;347(6229):1436-41.

58. Ludolph A, Drory V, Hardiman O, Nakano I, Ravits J, Robberecht W, Shefner J; WFN Research Group On ALS/MND. A revision of the El Escorial criteria - 2015. Amyotroph Lateral Scler Frontotemporal Degener. 2015 Jun 29:1-2.

59. Smith R, Myers K, Ravits J, Bowser R: Amyotrophic lateral sclerosis: Is the spinal fluid pathway involved in seeding and spread? Med Hypotheses. 2015 Jul 20. pii: S0306-9877(15)00278-9. PMID: 26220261

60. Emde A, Liu L-L, Libby RT, Reichenstein I, Oppenheim H, Eilam R, Silvestroni A, Alajajian B, Bosco DA, Sons R, Aebischer J, Schneider B, Hammond SM, Ravits JM, Möller T*, Hornstein E* (shared contributions): microRNA dysregulation in amyotrophic lateral sclerosis. EMBO J. 2015 Sep 1. pii: e201490493. PMID: 26330466.

61. Lasiene J, Komine O, Fujimori-Tonou N, Powers B, Endo F, Watanabe S, Shijie J, Ravits J, Horner P, Misawa H, Yamanaka K: Neuregulin 1 confers neuroprotection in SOD1-linked amyotrophic lateral sclerosis mice via restoration of C-boutons of spinal motor neurons. Acta Neuropathologica Communications Acta Neuropathol Commun. 2016 Feb 18;4(1):15. doi: 10.1186/s40478-016-0286-7. PMID: 26891847

62. Sellers DL, Bergen JM, Johnson RN, Back H, Ravits JM, Horner PJ, Pun SH.: Targeted axonal import (TAxI) peptide delivers functional proteins into spinal cord motor neurons after peripheral administration. Proc Natl Acad Sci U S A. 2016 Mar 1;113(9):2514-9. doi: 10.1073/pnas.1515526113. Epub 2016 Feb 17. PMID: 26888285

63. Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C. Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs. Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21. PMID: 27112497


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64. Kapeli K, Pratt GA, Vu AQ, Hutt KR, Martinez FJ, Sundararaman B, Batra R, Freese P, Lambert NJ, Huelga SC, Chun SJ, Liang TY, Chang J, Donohue JP, Shiue L, Zhang J, Zhu H, Cambi F, Kasarskis E, Hoon S, Ares M J, Burge CB, Ravits J, Rigo F, Yeo GW: Distinct and shared functions of ALS-associated proteins TDP-43, FUS and TAF15 revealed by multisystem analyses. Nat Commun. 2016 Jul 5;7:12143. doi: 10.1038/ncomms12143.

65. Ito Y, Ofengeim D, Najafov A, Das S, Saberi S, Li Y, Hitomi J, Zhu H, Chen H, Mayo L, Geng J, Amin P, DeWitt JP, Mookhtiar AK, Florez M, Ouchida AT, Fan JB, Pasparakis M, Kelliher MA, Ravits J, Yuan J. RIPK1 mediates axonal degeneration by promoting inflammation and necroptosis in ALS. Science. 2016 Aug 5;353(6299):603-8. doi: 10.1126/science.aaf6803. PMID: 27493188

66. Braak H, Albert C. Ludolph AC, Neumann M, Ravits J*, Del Tredici K* (*equal contributions): Pathological TDP-43 changes in Betz cells differ from those in bulbar and spinal alpha-motoneurons in sporadic amyotrophic lateral sclerosis. Acta Neuropathol. 2017 Jan;133(1):79-90. doi: 10.1007/s00401-016-1633-2. PMID:27757524.

67. Orr JE, Deacon N, Ravits J.: Sleep apnea in familial dysautonomia: A reflection of apnea pathogenesis. J Clin Sleep Med. 2016 Dec 15;12(12):1583-1584. doi: 10.5664/jcsm.6334. PMID:27855745.

68. Williams SM, Khan G, Harris BT, Ravits J, Sierks MR. TDP-43 protein variants as biomarkers in amyotrophic lateral sclerosis. BMC Neurosci. 2017 Jan 25;18(1):20. doi: 10.1186/s12868-017-0334-7. PMID: 28122516

69. Da Cruz S, Bui A, Saberi S, Lee SK, Stauffer J, McAlonis-Downes M, Schulte D, Pizzo DP, Parone PA, Cleveland DW*, Ravits J* (*equal contributions): Misfolded SOD1 is not a primary component of sporadic ALS. Acta Neuropathol. 2017 Feb 28. doi: 10.1007/s00401-017-1688-8. PMID:28247063.

70. Ditsworth D, Maldonado M, McAlonis-Downes M, Sun S, Seelman A, Drenner K, Arnold E, Ling SC, Pizzo D, Ravits J, Cleveland DW, Da Cruz S.: Mutant TDP-43 within motor neurons drives disease onset but not progression in amyotrophic lateral sclerosis. Acta Neuropathol. 2017 Mar 29. doi: 10.1007/s00401-017-1698-6. PMID: 28357566

71. Kawamata H, Peixoto P, Konrad C, Palomo G, Bredvik K, Gerges M, Valsecchi F, Petrucelli L, Ravits JM, Starkov A, Manfredi G.: Mutant TDP-43 does not impair mitorchondrial bioenergetics in vitro and in vivo. Mol Neurodegener. 2017 May 8;12(1):37. doi: 10.1186/s13024-017-0180-1

BOOK CHAPTERS 1. McDonald WI (eds): Diseases of the Nervous System. Ardmore Medical Books and William

Heinemann Medical Books, 1986. 2. Patterson L, Ravits J: Myasthenia gravis. In: Taylor RD (ed): Difficult Medical Management.

WB Saunders Co., 1991. 3. Ravits J: Muscle weakness. In: Fihn SD and McGee SR (eds): Outpatient Medicine. WB

Saunders Co., 1992 and 1998 (2nd edition). 4. Ravits J: Electromyography and nerve conduction studies. In: Fihn SD and McGee SR

(eds): Outpatient Medicine. WB Saunders, Co., 1992 and 1998 (2nd edition). 5. Ravits J, Laurie P, and Stone B: Amyotrophic lateral sclerosis microgenomics. Phys Med

Rehabil Clin N Am. 2005;16 (4):909-24. 6. Ravits J, Traynor BJ: Current and future direction in genomics of amyotrophic lateral

sclerosis. Phys Med Rehabil Clin N Am 2008;19(3):461-477. 7. Ravits J: Focal mononeuropathy onset of amyotrophic lateral sclerosis. Peripheral

Neuropathy. In Companion to Peripheral Neuropathy, Illustrated Cases and New Developments, Dyck PJ et al (eds), Saunders Elsevier, Philadelphia, 2010, ch 37, pp 167-171.


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8. Bruijn L and Ravits J: Convergence and Divergence in ALS Syndrome: Genetics, Molecular Pathology and Molecular Mechanisms. In: Motor Neuron Disease in Adults, Bromberg MB (ed), Oxford University Press, Oxford, 2015, chapter 16, pp 174-189.

9. Saberi S, Stauffer J, Schulte D, and Ravits J: Neuropathology of ALS and its variants. Neurologic Clinics Neurol Clin. 2015 Nov;33(4):855-76. doi: 10.1016/j.ncl.2015.07.012. Review.

10. Grad LI, Rouleau GA, Ravits J* and Cashman NR* (* co-corresponding authors): Clinical spectrum of amyotrophic lateral sclerosis (ALS). Cold Spring Harb Perspect Med. 2016 Dec 21. pii: a024117. doi: 10.1101/cshperspect.a024117. PMID: 28003278

GROUP AUTHORED (formally acknowledged in group authorship

1. The ALSUntangled Group. ALSUntangled Update 1: investigating a bug (Lyme disease) and a drug (Iplex). Amyotrophic Lateral Sclerosis 2009;10:248-250.

2. The ALSUntangled Group. ALSUntangled Update 2: investigating the Hickey Wellness Center. Amyotrophic Lateral Sclerosis 2009;10:490-491.

3. The ALSUntangled Group. ALSUntangled Update 3: investigating stem cell transplants at the hospital san jose tecnologico de monterrey. Amyotrophic Lateral Sclerosis 2010;11:250-252.

4. The ALSUntangled Group. ALSUntangled Update 4: investigating the xcell-center. Amyotrophic Lateral Sclerosis 2010;11:337-338.

5. The ALSUntangled Group. ALSUntangled Update 5: investigating the Stowe/Morales ALS Protocol. Amyotrophic Lateral Sclerosis 2010;11:414-416.

6. The ALSUntangled Group. ALSUntangled Update 6: investigating Marty Murray’s Method. Amyotrophic Lateral Sclerosis 2010;11:494-496.

7. The ALSUntangled Group. ALS Untangled No. 7: Investigating hyperimmune goat serum (Aimspro) for ALS. Amyotrophic Lateral Sclerosis 2010;11:571-573.

8. The ALSUntangled Group. ALSUntangled No. 8: Low dose naltrexone for ALS. Amyotrophic Lateral Sclerosis 2011;12:76-78.

9. The ALSUntangled Group. ALSUntangled No. 9: blue green algae (Spirulina) as a treatment for ALS. Amyotrophic Lateral Sclerosis 2011;12:153-155.

10. The ALSUntangled Group. ALSUntangled No. 10: luteolin and lutimax. Amyotrophic Lateral Sclerosis 2011;12:235-237.

11. The ALSUntangled Group. ALSUntangled No. 11: NuTech Mediworld. Amyotrophic Lateral Sclerosis 2011;12:309-311.

12. The ALSUntangled Group. ALSUntangled No. 12: Dean Kraft, Energy Healer. Amyotrophic Lateral Sclerosis 2011;12:389-391.

13. The ALSUntangled Group. ALSUntangled No. 13: Bee Venom. Amyotrophic Lateral Sclerosis 2011;12:471-472.

14. The ALSUntangled Group. ALSUntangled No. 14: Mototab Amyotrophic Lateral Sclerosis 2012;13:161-164.

15. The ALSUntangled Group. ALSUntangled No. 15: Coconut Oil. Amyotrophic Lateral Sclerosis 2012;13:328-330.

16. The ALSUntangled Group. ALSUntangled No. 16: Cannabis. Amyotrophic Lateral Sclerosis 2012;13:400-404.

17. The ALSUntangled Group. ALSUntangled No. 17: “When ALS Is Lyme.” Amyotrophic Lateral Sclerosis 2012;13:487-491.

18. The ALSUntangled Group. ALSUntangled No. 18: Apoaequorin (Prevagen). Amyotroph Lateral Scler Frontotemporal Degener. 2013 Jan;14(1):78-9.

19. ALSUntangled Group. ALSUntangled No. 19: Sodium chlorite. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Apr;14(3):236-8.

20. ALSUntangled Group. ALS Untangled No. 20: the Deanna protocol. Amyotroph Lateral Scler Frontotemporal Degener. 2013 May;14(4):319-23.


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21. ALS Untangled No. 21: Fecal transplants. ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Sep;14(5-6):482-5. PMID: 23870000

22. ALS Untangled No. 22:. Propofol. ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Dec;14(7-8):640-2. doi: 10.3109/21678421.2013.826469.

23. ALS Untangled No. 23: The Rife machine and retroviruses. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Mar;15(1-2):157-9. doi: 10.3109/21678421.2013.850802

24. ALS Untangled No. 24: Vitamin D. ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Jun;15(3-4):318-20. doi: 10.3109/21678421.2014.888871

25. ALS Untangled No. 25: Ursodiol . ALSUntangled Group. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):475-8. doi: 10.3109/21678421.2014.931010

26. ALSUntangled No. 27: Precision Stem Cell. Amyotroph Lateral Scler Frontotemporal Degener. 2015 Jun;16(3-4):282-5. doi: 10.3109/21678421.2015.1024571. Epub 2015 Mar 26. PMID: 25807846

27. ALSUntangled Group. ALSUntangled No. 28: Acupuncture. Amyotroph Lateral Scler Frontotemporal Degener. 2015 Jun;16(3-4):286-9. doi: 10.3109/21678421.2015.1039240. Epub 2015 May 11. PMID: 25960084.

28. ALSUntangled Group. ALSUntangled No. 29: MitoQ. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):427-9. doi: 10.3109/21678421.2015.1050897. PMID: 26090901

29. ALSUntangled Group. ALSUntangled No. 30: methylcobalamin.. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(7-8):536-9. doi: 10.3109/21678421.2015.1070574. Epub 2015 Jul 23. PMID: 26203660

30. ALSUntangled Group. ALSUntangled No. 31: Protandim. Amyotroph Lateral Scler Frontotemporal Degener. 2015;17(1-2):154-6. PMID: 26414415

31. ALSUntangled Group. ALSUntangled No. 32: Gluten-free diet. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17(3-4):307-9. PMID: 26473662

32. ALSUntangled Group. ALSUntangled No.33 Endotherapia. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):461-5. PMID: 26808361

33. ALSUntangled Group. ALSUntangled No. 34: GM604. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Apr 4:1-5. PMID: 27043278

34. ALSUntangled Group. ALSUntangled No. 35: Hyperbaric Oxygen Therapy. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Apr 18:1-3. PMID: 27089115

35. ALSUntangled Group. ALSUntangled No. 36: Accilion. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Aug 9:1-6. PMID: 27685025

36. ALSUntangled Group. ALSUntangled No. 37: Inosine. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Aug 30:1-4. PMID: 27575981

37. ALSUntangled Group ALSUntangled 38: ????? Amyotroph Lateral Scler Frontotemporal Degener.

38. ALSUntangled Group ALSUntangled 39: Acuscope (micro-Amp electrical muscle stimulation) Amyotroph Lateral Scler Frontotemporal Degener. Pages: 1-4 | DOI: 10.1080/03007995.2017.1285468

ABSTRACTS (selected)

1. Ravits J, Baker M, Wilkins D, Hallett M. Primary Writing Tremor and Myoclonic Writer’s Cramp. Muscle Nerve. 1984 Sep;7(7):578-579

2. Ravits J, Baker M, Wilkins D, Hallett M. Spontaneous Reticular Myoclonus. Muscle Nerve. 1985 Sep;8(7):621

3. Ravits J, Baker M, Wilkins D, Hallett M, Nilsson J, Brown R, Polinsksy R. A Comprehensive Study of Electrophysiologic Tests of Autonomic Function in Patients with Primary Autonomic Failure. Muscle Nerve. 1986 Sep;9(7):657

4. Ravits J, Hallett M, Baker M, Wilkins D: Adult-onset body tic. Neurology 1986; 36 (suppl. 1):118


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5. Baker M, Hallett M, Wilkins D, Ravits J, et al: Physiology of epilepsia partialis continua. Neurology 1986; 36 (suppl.1):128

6. Ravits JM, Buchthal F, Barton N. Clinical and Electrophysiologic Findings in Fabry’s Disease. Muscle Nerve. 1990 Sep;13(9):855

7. Ravits J, Laurie P, Stone B: Motor Neuron Isolation by Tissue Microdissection for Biochemistry in ALS Nervous Systems. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2004;5 (Suppl 2), 71.

8. Laurie P, Ravits J, Stone B: RNA Assessment in ALS Nervous Systems and Motor Neurons Isolated by Microdissection in ALS Nervous Systems. Amyotrophic Lateral Sclerosis and other motor neuron disorders 2004;5 (Suppl 2), 71-72.

9. Ravits J, Laurie P, Fan Y: Focality of ALS: 3-D Topography of Lower Motor Neuron Degeneration in Post-Mortem Nervous Systems and the Neuropathologic Gradient of Degeneration. Neurology 2006;66(5,Suppl 2):A244-A2245.

10. Ravits J, Paul P, Jorg C: ALS at the Onset: Topography of UMN and LMN Loss. Amyotrophic Lateral Sclerosis 2006;7 (Suppl 1),11.

11. Ravits J, Fan Y, Laurie P, Stone B, Bumgarner R: Differential Genes Set Expression in SALS Motor Neuron and Anterior Horn Enriched RNA Pools Using Whole Genome Oligonucleotide Microarray. Amyotrophic Lateral Sclerosis 2007;8 (Suppl 1),11-12.

12. Ravits J, Laurie P, Fan Y, Stone B. Correlation Between Genome-wide SNP and Whole Genome Expression: Discovery-based Pursuit of SALS Pathobiology. Amyotrophic Lateral Sclerosis 2007;8 (Suppl 1),12.

13. Ravits J, Fan Y, Rabin S, Stone B, Beyer D, Bammler T, Bumgarner R, La Spada A: Differential Genes Set Expression in G93A Transgenic Mice Using Whole Genome Oligonucleotide Microarray. Amyotrophic Lateral Sclerosis 2008;9 (Suppl 1),3.

14. Ravits J, Laurie P, Kim Y: Distribution of TDP-43 Intracytoplasmic Inclusions in the ALS Gradient of Neurodegeneration. Amyotrophic Lateral Sclerosis 2008;9 (Suppl 1),15.

15. Leverenz, J, Bird, T, Rumbaugh, M, Yu, C, Cruchaga, C, Steinbart, E, and Ravits J: Is the Arg5His MAPT variant pathogenic for dementia and motor neuron disease? Alzheimer’s & Dementia – Alzheimer’s Dement 01/2011;7(4). DOI:10.1016/j.jalz.2011.05.569

16. Song F, Chiang P, Ravits J, Loeb J: Abnormal axoglial communication in the corticospinal tracts could underlie upper motor neuron degeneration in sporadic ALS. Amyotrophic Lateral Sclerosis 2011;12(suppl 1):3.

17. Song F, Chiang P, Ravits J, Loeb J: Aberrant neuregulin signaling in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis 2011;12(suppl 1):115.

18. Baughn, M, Sun S, Lagier-Tourenne C, Pizzo D, Simpkinson M, Baloh R, Vandenberg S, Cleveland D, Ravits J. MNDA International Symposium on ALS/MND 2012. Nuclear RNA foci in hexanucleotide expanded C9FTD/ALS. Amyotrophic Lateral Sclerosis 2012 13:S1, 99-110.

19. Song F, Liu J, Ravits, J, Loeb J: Size-dependent axon loss in the corticospinal tract in ALS patients with upper motor neuron signs. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2013;14(suppl 2),2.

20. Sareen D, O'Rourke G, Muhammad AK, Grant S, Simpkinson M, Bell S, Carmona S, Ornelas L, Sahabian A, Baughn M, Ravits J, Harms MB, Rigo F, Bennett CF, Svendsen CN, Baloh RH: Targeting RNA foci shows a therapeutic effect in iPSC-derived motor neurons from C9ORF72 repeat patients. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2013;14(suppl 2),16.

21. Lagier-Tourenne C, Baughn, M, Riggo, F, Sun S, Markmiller S, Watt, A, Sun Y, Polymenidou M, Qui J, Li, H, Xue Y, Chun, S, Liu, P, Katz, M, Freier, S, Fu XD, Yeo GW, Bennett, F, Cleveland DW, Ravits J: Therapy development for ALS/MND and frontotemporal dementia with C9orf72 expansion: Antisense oligonucleotide mediated reduction in nuclear RNA foci. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2013;14(suppl 2),17.


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22. Hutt, K, Rabin, S, Baughn, M, Libby, R, Markmiller, S, Hoon, S, Yeo, GW, and Ravits J: More evidence supporting perturbation in extracellular and transmembrane domains and of protein signaling by transcriptome analysis of motor neurons from sporadic ALS spinal cords. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2013;14(suppl 2),35.

23. Lagier-Tourenne C, Saberi S, Jiang J, Zhu Q, Gendron TF, Jafar-nejad P, Stauffer J, Schulte D, Ling S-C, Van Damme P, Shaw C, Bennett CF, Petrucelli L, Da Cruz S, Rigo F, Ravits J, Cleveland D: Gain of Toxicity From Hexanucleotide Expansion in C9orf72 in ALS and Frontotemporal Dementia Is Alleviated By Antisense Oligonucleotides Target Repeat-Containing RNAs. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2015;16(suppl 1), 2.

24. Jiang J, Zhu Q, Gendron TF, Rigo F, Da Cruz S, Mcalonis-Downes M, Saberi S, Drenner K, Schulte D, Sun Y, De Muynck L, Ling S-C, De Jong P, Edbauer D, Shaw C, Bennett CF, Petrucelli L, Ravits J, Cleveland D, Lagier-Tourenne C. A Gain of Toxicity by C9orf72 GGGGCC Expansion in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2015;16(suppl 1), 15.

25. Ravits J. Understanding Disease Progression in ALS. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2015;16(suppl 1), 24.

26. Drawert B, Thakore N, Mitchell B, Pioro E, Ravits J, and Petzold L: Modeling neuroanatomic propagation of ALS in the spinal cord. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2016;17(suppl 1), 16-7.

27. Song F, Dachet F, Liu J, Rabin S, and Ravits J: Predicting novel relationship of the caudal distribution of lower motoneurons in relation to the region of clinical onset to their connected glia in sporadic ALS patients. Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration 2016;17(suppl 1), 63-4.

john m. ravits, md - uc san diego school of medicine€¦ · · 2017-07-20american board of...

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