advocacy day 2014 washington, d.c. march 4-5, 2014 · advocacy day 2014 washington, d.c. march 4-5,...
TRANSCRIPT
ADVOCACY DAY 2014
WASHINGTON, D.C.
March 4-5, 2014
1615 Bonanza St., Suite 202 Walnut Creek, CA 94596
(800) 688-8765 www.fragilex.org
“One Person Can Make A Difference and Everyone Should Try” John F. Kennedy
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Table of Contents
SECTION I: Advocacy Day Agenda
SECTION II: What We Are Doing Here and Why it’s Important
SECTION III: The Substance of our Ask – What, Why and How
SECTION IV: Preparing for Your Hill Visits – Hook, Line and Sinker
SECTION V: Role Play and Practice Sessions
SECTION VI: Supplemental Information
Article: New Roadmap to Unlock the Mystery of Autism: Is Fragile X the Key? Some Deeper Dive Talking Points Frequently Asked Questions Likely Questions from Congressional Staff Samples: Dear Colleague and Appropriations Committee Letters
SECTION VII: What to Do When You Go Home
Completing the Meeting Report Forms
Sending Thank You letters and Personal Notes
SECTION VIII: Contact Information
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SECTION I: Advocacy Day Agenda
Tuesday March 4, 2014
Training Agenda
Noon – 12:45pm Registration (sign in and pick up materials)
1:00pm – 1:15pm Welcome to Advocacy Day 2014
Brad Whitus, NFXF Board of Directors President and Jimi
Grande, NFXF Board of Directors and Chair of Public
Policy & Government Affairs Committee
1:15pm – 1:30pm What We Are Doing Here and Why it’s Important
Jeffrey Cohen, NFXF Interim Executive Director and Director
of Government Affairs and Advocacy
1:30pm – 2:15pm The Substance of Our Ask - What, Why and How
Jeffrey Cohen and Jimi Grande
2:15pm – 2:30pm Break
2:30 – 3:45pm Interactive Hill Preparation
Christopher Kush, CEO, Soapbox Consulting
3:45 to 4:00pm Break and Schedules Distributed
4:00 – 5:00 State Breakouts and Practice Sessions
Holly Roos, NFXF Community Support Network Coordinator
and Denise Devine, FX Resource Group of Western
Massachusetts Leader
5:00 – 6:00 Socialize and Cash Bar
Soapbox Help Desk Open for Questions and Scheduling
Matters
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Wednesday March 5, 2014
Capitol Hill Visits
6:00am – 6:30am Sheraton Four Points Hotel Lobby
Pre-Ordered A la carte Breakfast for Purchase
6:30am – 6:45am Board Buses for Trip to Capitol Hill Steps
7:15am - 7:30pm Convene on East Capitol Hill Steps
Photo and Security Clearance for House Chambers Tour
8:00am – 9:00am House Chambers Tour with Congressman Gregg Harper
9:30am – 5:00pm Congressional Hill Visits
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Section II: What Are We Doing Here and Why
The Federal Budget Process The Role of our Advocacy Program in the Budgeting Process
Our Senate and House Champions
Dear Colleague and Sign-on Letters
Each year our Senate and House champions send a letter to their colleagues (Dear Colleague letter) asking them to support FX by signing-on to a Letter to Appropriators which asks the Appropriations Committee to accept our proposed Appropriations Language in next year’s budget. YOU, ask YOUR Members of Congress (MOC) to SIGN THE LETTER. After collecting as many signatures as we can, our champions send the letter to the Appropriations Committee for their consideration before next year’s budget is finalized. So in your meetings when you ask your MOC to “sign the letter” or “sign on” you’re asking them to put their names on the letter to Appropriators. Sample letters are included in Section VI so you can see what one looks like. These letters are also in the Leave-Behind Packets so your MOC can review what you’re asking them to sign.
“I’m Just a Bill” - Video
Our Goals:
1. Sustain federal investments in translational research and drug development (NIH and DOD) and in dedicated public health initiatives and data collection (CDC NCBDDD) at not less than current levels of funding.
2. Require federal agencies with fragile x and autism portfolios to combine efforts and resources to better understand the shared science between the two and bring effective treatments for both conditions to market. This includes continued implementation of the NIH Research Plan for Fragile X, implementation of the new CDC NCBDDD Public Health Research Plan for Fragile X and continued federal support for the Fragile X Clinical & Research Consortium.
3. Pass Bills that will have an immediate effect on the quality of life of individuals living with Fragile
X-associated Disorders and their families. This includes the Achieving a Better Life Experience (ABLE) Act to create tax-advantaged savings plans for individuals with disabilities.
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The Reasons Behind Our Ask: So you know why we’re asking for what we are
and so you’re prepared if you have the opportunity to engage is a more detailed
discussion with your MOC or their staff…….
-WE’RE CLOSE TO TARGETED DRUG TREATMENTS : The mechanism in the brain that causes FXS is known. Drugs that have reversed this mechanism in animals are now in human trials. The same protein, which when missing causes FXS, controls hundreds of genes believed to cause autism. Drugs that can treat FX will likely benefit those with autism and vice versa. Treatments that can reverse the symptoms of FX will likely require a combination of drugs and figuring out which combinations work best for who and at what dose will take time, but WE ARE WELL ON OUR WAY. Congress has supported groundbreaking work at the NIH, CDC and DOD that has made this possible. To turn back now would have a devastating effect on our ability to complete this work. For these reasons, we are asking Congress to continue (and -- when possible -- to grow) federal investments in translational research at the NIH and DOD. Because of already reduced funding, federal agencies with separate FX and autism research portfolios must explore ways to combine efforts and resources to get the most out of limited resources, accelerate the pace of research and shorten the time to bring treatments for these interrelated conditions to market -THE PUBLIC HEALTH IMPLICATIONS OF FX ARE MORE SIGNIFICANT THAN PREVIOUSLY REALIZED. Longitudinal data gathered by CDC supported research is shedding new light on the significant impact of FX mutations. New treatments will create an instant and potentially overwhelming demand for access to care across the populations impacted by both FX and autism. We must build upon important work already made possible by Congress and sustain federal Public Health investments in the CDC’s Fragile X Public Health initiative. Through the National Center on Birth Defects and Developmental Disabilities, Congress has invested significantly in the creation of the Fragile X Clinical & Research Consortium (FXCRC), a network of 26 clinics across the country that are collecting vital public health data, creating a patient database and registry which will identify treatment targets and support clinical trials of targeted drug therapies, and compile best practices for the treatment of Fragile X-associated Disorders. To turn back now would have a devastating effect on the ability to complete this work. For these reasons, we are asking Congress to continue (and when possible to grow) federal investments in public health research at the CDC to fully understand the implications of the FX mutation and ensure access to therapeutic services and supports through continued funding. Likewise here, branches and divisions with separate FX and autism research portfolios must explore ways to combine efforts and resources to get the most out of limited resources.
-INDIVIDUALS WITH FXS, AUTISM AND OTHER IDDs SEEK THE SAME OPPORTUNITES TO REACH MAXIMUM SELF-SUFFICIENCY THAT OTHERS ALREADY ENJOY: Individuals with intellectual disabilities like FXS and autism are capable and motivated to attain a greater degree of self-sufficiency than current laws allow. They want to work and save and their families want to help them reach the highest level of self-sufficiency possible but antiquated asset limitations, all-or-nothing restrictions on other government benefits and the absence of tax incentivized savings programs that all other enjoy make the transition to adult life difficult often resulting in a life of isolation and dependence.
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For these reasons, we are asking Congress to pass the ABLE Act which will allow individuals and their families to save money without losing critically needed supports and to attain optimal economic self-sufficiency.
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Section III: The Substance of our Ask
During your Congressional visits
The OPENING Introduce yourself and be sure to include the name of your city;
Explain that you are in Washington with nearly 200 others from across the country for the 11th
Annual NFXF Advocacy Day
Explain your connection to FX including the name of your family member impacted (show picture);
Have a short anecdote ready that makes a powerful point about how your life/family has been
impacted;
Note: Everyone on your team should have an opportunity to introduce themselves.
From there you can delegate different parts of your presentation to different team
members and switch roles from meeting to meeting. When you meet with your
group throughout the day on Tuesday talk about how you want to divide the
presentation.
The ASK: Remember: We are only asking for 3 things
1. SIGN THE LETTER to Appropriators that is being circulated by our champions
This ask pertains to our request for Directive Appropriations Language (DAL)
We hope the Representative/Senator will support key federal programs focused on Fragile X-associated Disorders. Specifically, we ask that you SIGN THE LETTER to Appropriators that is being circulated by our champions which asks that they:
Maintain dedicated support for CDC’s national Fragile X public health program and continue to support the Fragile X Clinical & Research Consortium.
Create greater efficiency and synergy among the FX and autism research tracks at NIH, CDC and DOD to accelerate translational research toward a better understanding of both conditions and shorten the time to bring effective treatments for both conditions to market.
Support for the continued inclusion of Fragile X-associated Disorders among the list of eligible healthcare conditions for targeted biomedical research funding through the U.S. Department of Defense Peer Reviewed Medical Research Program.
If you say nothing else, you need to say……… “Please sign our Champion’s Letter to Appropriators-a copy is in the leave behind folder.”
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2. SIGN THE LETTER to the House Energy and-& Commerce Committee that is being circulated by our champions
This ask pertains to our request for a hearing before the committee
HOUSE OFFICES
Request that your Representative sign onto a letter to the House Energy and-& Commerce Committee – urging them to convene a hearing on the intersections between Fragile X and autism.
o We want to ensure the Department of Health & Human Services (HHS), the National Institutes of Health (NIH), industry, and other key stakeholders are providing appropriate investments in these areas and believe a Congressional hearing in the House Energy & Commerce Committee focused on these intersections would yield significant attention to these important issues for people with Fragile X Associated Disorders and autism
To sign onto this letter (see example in your leave behind packets), please contact Scot Malvaney in Congressman Gregg Harper’s office at [email protected]
SENATE OFFICES
As an FYI, mention to Senate offices that we are pushing for a hearing on this topic to House Energy & Commerce Leadership. If you say nothing else, you need to say……… “Please sign the letter to the House Energy and Commerce Committee supporting our request for a hearing on the intersections between Fragile X and autism.
3. Help us pass the ABLE Act in the 113th Congress
This ask pertains to our request to pass the ABLE Act
We hope the Representative/Senator will help us get the ABLE Act passed in the 113th Congress.
Over 350 Members of Congress in both chambers have already co-sponsored this Bill. The ABLE Act
will have an immediate impact on the life of my (son/daughter/family member/family) impacted
by a Fragile X-associated Disorder and will allow me to begin saving for his/her future and help
him/her reach the highest level of self-sufficiency and independence possible.
ASK: COSPONSOR
If Member is already a cosponsor, thank them for their support of the bill and remind them that there over 400 Members of Congress also supporting this bill.
If they are NOT a cosponsor, please ask them to cosponsor the bill and tell them why it’s important to our community.
o If the Member is interested in cosponsoring, they should contact: HOUSE: Jennifer Debes in Rep. Crenshaw’s office ([email protected]) SENATE: Jennifer McCloskey in Sen. Casey’s office
([email protected]) SIGN ONTO LETTER
Given the widespread support of the ABLE Act, request that your Member sign onto our letter to House/Senate Leadership which requests that the ABLE Act be brought to the House/Senate Floor for a vote as soon as possible.
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o If the Member is interested in signing on, they should contact the following offices by March 7th:
HOUSE: Jennifer Debes in Rep. Crenshaw’s office ([email protected]) SENATE: Jennifer McCloskey in Sen. Casey’s office
If you say nothing else, you need to say……… “Please contact the offices of the ABLE Act’s
champions and let them know that you will support and vote for passage of the bill in this
Congress.
The CLOSE
Follow-up and Thank You
Can we count on the Representative’s/Senator’s support for these requests? May I follow up with you once you’ve had a chance to consider our requests or discuss them with your boss? How should I get in touch with you? (Be sure to get the card of the individual you meet with so you have their contact information)
THANK YOU for your time and consideration of our requests. It means a great deal to me to know that you care about my family and our priorities. For me, for my family and for all the families impacted by a FXD back home in our community and across our state…Thank You.
(If you met with a member of the staff) I’d like to follow up directly with the Senator/Representative next time he/she is in the home state office. There are other families that couldn’t make the trip to DC and many would like to meet at your local office. Who should I contact to set that up?
A solid handshake and a warm smile go a long way toward sealing the deal!
Now would be a perfect time to take a minute to fill out the Meeting Evaluation
Form. You can enter the information from a smartphone, tablet or laptop or just
make notes on the paper form provided, but capture this information while it’s
fresh in your mind. If you’re rushed to get to your next meeting take the first
opportunity you have.
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Section IV: Preparing for Your Hill Visits
Christopher Kush, MPP, CEO/Author
113TH Congress by Party
Parts of a Legislative Meeting
Hook
Line
Sinker
Tips for Handling Push-Back
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Section V: Role Play and Practice Sessions
The Importance of Practice
Divide and Conquer
Assigning Parts
Let’s Practice
Evaluate
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Section VI: Supplemental Information
Not Mandatory Reading but Good Background Material
New Roadmap to Unlock the Mystery of Autism: Is Fragile X the Key?
Genetic Research Breakthroughs Offering Hope for Treatment
Groundbreaking research and clinical trials are offering fresh hope to individuals and their families who are living with the closely linked conditions of fragile X syndrome (FXS) and autism. Because mutation of the fragile X (FMR1) gene is the most common known single gene cause of autism, scientific research that further links FXS to autism strengthens the case for a single focus for developing targeted medical treatments for both conditions.
Now, scientists have discovered additional, specific genetic links connecting the two conditions, a breakthrough that will likely lead to new treatments for both autism and FXS. These and other recent discoveries have already resulted in nationwide clinical trials of pharmaceuticals that could treat some of the core symptoms of both conditions, especially impaired social and communications skills and repetitive behaviors. Additional clinical trials, underway and planned, will give families more opportunities to participate, and will offer hope that their loved ones will ultimately benefit from these scientific advancements.
Background: The fragile X gene normally produces a protein – FMRP – which plays a critical role in regulating other genes and their proteins, many of which are needed for normal brain development and function. However, in FXS, that process is interrupted because the gene is turned off, causing the body to produce too little or no FMRP; the absence of which leads to a wide range or spectrum of symptoms, from intellectual disability to neurological and behavioral impairments. The overlap between autism and FXS is consequential. As many as three percent of those with autism with cognitive impairment have the fragile X mutation, but as many as sixty-seven percent of those with FXS are diagnosed with an autism spectrum disorder.
Taken together, recent research published in the medical journals Neuron, Nature and Science have shed considerable light on how FMRP plays a role in autism. Investigators now believe there are likely 350-400 “autism genes” and over 900 RNAs, which when mutated, cause autism. A significant percent (up to half) of these genes are regulated by FMRP in brain cells.
Potential: These investigators have identified a broad genetic roadmap linking the two conditions, giving researchers the ability to map the specific pathways that are disregulated in autism; such knowledge raises the prospect of more directly correcting the problems with individual pathways that arise in the absence of FMRP, thus facilitating the development of additional targeted treatments for autism and FXS. These findings could impact millions of Americans living with autism and FXS, and tens of millions worldwide.
In other ongoing research, scientists at medical centers throughout the U.S. and at multiple centers worldwide have been conducting human trials of a new drug developed by Seaside Therapeutics. Similar
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compounds developed or licensed by pharmaceutical giants Roche and Novartis are also in human trials. Researchers are studying the medications’ effects on social impairment in individuals with FXS and autism.
In early results, participants with severe social impairment showed a marked reduction of social avoidance, improvement in social functioning, and a reduction in problem behaviors. In earlier animal studies, key deficiencies in FXS and autism were reversed, correcting the lost communication between brain cells, as well as reducing seizures and repetitive behaviors (core symptoms of autism and FXS). According to a special report by Reuters, these treatment studies may reach beyond fragile X-related autism; the report notes that “early studies suggest the drugs may work in other forms of autism, too.”
Conclusion: As this work continues, the positive consequences for people with FXS and autism (and autism spectrum disorders) are substantial. There is great optimism for targeted medical treatments for many of the major symptoms of autism and FXS. New ways to more precisely identify the genetic roots of these conditions will lead to more accurate and earlier testing to identify affected individuals, and will provide the opportunity for more timely and effective interventions. Science is giving people with autism and fragile X syndrome, and their families, hope that such treatments are coming.
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Congressional Meeting Talking Points:
1. SIGN THE LETTER to Appropriators that is being circulated by our champions
This ask pertains to our request for Directive Appropriations Language (DAL)
We hope the Representative/Senator will support key federal programs focused on Fragile X-associated Disorders. Specifically, we ask that you SIGN THE LETTER to Appropriators that is being circulated by our champions which asks that they:
Maintain dedicated support for CDC’s national Fragile X public health program and continue to support the Fragile X Clinical & Research Consortium.
Create greater efficiency and synergy among the FX and autism research tracks at NIH, CDC and DOD to accelerate translational research toward a better understanding of both conditions and shorten the time to bring effective treatments for both conditions to market.
Support for the continued inclusion of Fragile X-associated Disorders among the list of eligible healthcare conditions for targeted biomedical research funding through the U.S. Department of Defense Peer Reviewed Medical Research Program.
2. House Energy & Commerce Hearing Background
Fragile X mutations affect ~1.5 million Americans. Only about 10% have Fragile X Syndrome but the remainder who have a pre-mutation (smaller mutation) are at risk of developing serious health conditions also related to the mutation.
o Fragile X and autism are two sides of the same coin. Fragile x has been called “one of the autisms”.
o The walls between FX and Autism are a function of artificial constructs not of scientific reality” and
o FX and autism share a relationship, the science that is important to FX is important to Autism and the science that is important to Autism is important to FX
Despite this reality funding streams at all agencies conducting research on both conditions remain for the most part –separate- and grossly disparate.
o Fragile X research has been leading the way toward drug treatments for FX and autism.
While the high incidence of autism is well established, scientists across many areas of interest are now taking note of its inextricable links to FX. At a time when autism is routinely referred to as an “epidemic” and more recently even a “pandemic” and a desperate search is underway to find its cause, the link to Fragile X likely holds the promise of the path forward toward finding an effective treatment for not only FX but autism as well.
o The Fragile X community is already working to build the necessary clinical tools and infrastructure
necessary for clinical trials through the CDC, NCBDDD funded Fragile X Clinical and Research Consortium (FXCRC) including a longitudinal database and patient registry.
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o Legislative Background/Information
Congress has already spoken on this issue. As recently as the DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, AND EDUCATION, AND RELATED AGENCIES APPROPRIATION BILL, 2014, The Committee commend[ed] NICHD for leading the effort to map the molecular, physiological, biological, and genetic connections between Fragile X (and the Fragile X protein) and autism and urge[d] all ICs with Fragile X and autism portfolios to explore these connections with the goals of better understanding both conditions and shortening the time it will take to bring effective treatments for both conditions to market.
We want to ensure the Department of Health & Human Services (HHS), the National Institutes of Health (NIH), industry, and other key stakeholders are providing appropriate investments in these areas and believe a Congressional hearing in the House Energy & Commerce Committee focused on these intersections would yield significant attention to these important issues for people with Fragile X Associated Disorders and autism.
ASK: HOUSE OFFICES
Request that your Representative sign onto a letter to the House Energy and-& Commerce Committee – urging them to convene a hearing on the intersections between Fragile X and autism.
o We want to ensure the Department of Health & Human Services (HHS), the National Institutes of Health (NIH), industry, and other key stakeholders are providing appropriate investments in these areas and believe a Congressional hearing in the House Energy & Commerce Committee focused on these intersections would yield significant attention to these important issues for people with Fragile X Associated Disorders and autism
To sign onto this letter (see example in your leave behind packets), please contact Scot Malvaney in Congressman Gregg Harper’s office at [email protected]
SENATE OFFICES
As an FYI, mention to Senate offices that we are pushing for a hearing on this topic to House Energy & Commerce Leadership.
3) Cosponsor/Sign onto the Achieving a Better Life Experience (ABLE) Act of 2013 (H.R. 647/S. 313)
Letter to House/Senate Leadership
Background – Why we need the ABLE Act?
Parents and individuals with disabilities need a savings tool to help them save for the future just as Americans in the broader population do. Developing assets is one way toward improving economic self-sufficiency. Savings will help people with disabilities to live full, productive lives in their communities.
The federal and state benefits system supplies critical services to people with disabilities but falls far short of what people need to live independently. At the same time, the system forces people into poverty in order to access services. There needs to be a flexible, portable, low cost savings tool to help supplement these short-falls.
Under current law, people with disabilities receiving federal benefits cannot have more than $2,000 in assets and must have a very low monthly income to remain eligible.
ABLE Act Ins & Outs
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The Achieving a Better Life Experience Act (ABLE) of 2013 (S. 313/H.R. 647) was introduced on Feb. 13th in the 113th Congress (2013-2014 Congressional cycle).
– The ABLE Act would amend Section 529 of the Internal Revenue Service Code of 1986 to create tax-free savings accounts for individuals with disabilities.
– The bill aims to ease financial strains faced by individuals with disabilities by providing a tax-free savings account that is low cost, portable, flexible, and protects their federal benefits.
– The funds can be used to supplement benefits provided through private insurances, the Medicaid program, the supplemental security income program, the beneficiary’s employment, and other sources.
An ABLE account could fund a variety of essential expenses for individuals, including medical and dental care, education, community based supports, employment training, assistive technology, housing, and transportation.
The ABLE Act provides individuals with disabilities the same types of flexible savings tools that all other Americans have through college savings accounts, health savings accounts, and individual retirement accounts.
The legislation also contains Medicaid fraud protection against abuse and a Medicaid pay-back provision when the beneficiary passes away. It will eliminate barriers to work and saving by preventing dollars saved through ABLE accounts from counting against an individual’s eligibility for any federal benefits program.
With an ABLE account, the beneficiary’s SSI check would be suspended during any period of time the account has assets over $100,000. It is important to note that although the payment is suspended the individual does not lose their eligibility to receive the payment, so that when the assets are spent down it can be reinstated.
Congressional Background Information
This is the fourth Congress into which the ABLE Act has been introduced. There are nearly 400 Members of Congress supporting this bipartisan bill – 64 Senators and 332 Representatives.
The bill is being led by a bipartisan, bicameral set of Congressional champions, including: o Senator Robert Casey, Jr., (D-PA) and Senator Richard Burr (R-NC) o Congressman Ander Crenshaw (R-FL), Congressman Chris Van Hollen (D-MD),
Congresswoman Cathy McMorris Rodgers (R-WA) and Congressman Pete Sessions (R-TX)
The bill has been scored by the Joint Tax Committee (JCT) and awaiting the Congressional Budget Office (CBO) Score. If offices have specific questions about the score, please let Jeffrey Cohen ([email protected]) know and he will follow up.
Because of the wide-bipartisan, bicameral support, we want the ABLE Act brought to the House/Senate Floor for a vote as soon as possible.
ASK: (Your schedule will indicate whether your MOC has already co-sponsored) COSPONSOR
If Member is already a cosponsor, thank them for their support of the bill and remind them that there over 400 Members of Congress also supporting this bill.
If they are NOT a cosponsor, please ask them to cosponsor the bill and tell them why it’s important to our community.
o If the Member is interested in cosponsoring, they should contact: HOUSE: Jennifer Debes in Rep. Crenshaw’s office ([email protected])
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SENATE: Jennifer McCloskey in Sen. Casey’s office ([email protected])
SIGN ONTO LETTER
Given the widespread support of the ABLE Act, request that your Member sign onto our letter to House/Senate Leadership which requests that the ABLE Act be brought to the House/Senate Floor for a vote as soon as possible.
o If the Member is interested in signing on, they should contact the following offices by March 7th:
HOUSE: Jennifer Debes in Rep. Crenshaw’s office ([email protected]) SENATE: Jennifer McCloskey in Sen. Casey’s office
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Frequently Asked Questions: 1. How many people have a mutation of their Fragile X gene (are Fragile X carriers)?
Nearly 1,500,000
2012 studies funded by the CDC and NIH estimated that 1 in 151 females and 1 in 468 males in the United
States may be affected by a fragile X permutation. These are the first population based studies which have
begun to more accurately characterize the true prevalence of FX mutations and that number is
substantially higher than thought before. Using these numbers and the current US population you can say
that as many as 1,500,000 in the US are carriers of a mutation of their FX gene. People with a fragile X
premutation do not have fragile X syndrome but might have another fragile X-associated disorder. Some
people with fragile X premutations have noticeable symptoms, and others do not. These statistics are
important because both men and women are at risk for having symptoms linked to fragile X-associated
disorders.
o Women with a premutation reported their last menstrual cycle at an earlier age than women without a premutation (48 vs. 51 years).
o Men and women with a premutation were more than four times as likely to report dizziness or fainting as people without a premutation (18% vs. 4%). Men and women with a premutation were more than twice as likely to report numbness as people without a premutation (29% vs. 13%).
2. How many people in the US have Fragile X Syndrome?
Nearly 100,000.
Estimates for males and females with a full mutation of their Fragile x gene range from 1 in 3600 to
1 in 5000. Using these numbers and the current US population you can say that as many as
100,000 individuals in the US have Fragile x Syndrome. In a CDC funded national survey:
Nearly all males with FXS had developmental delay or intellectual disability (96%)
Nearly 2/3 of females with FXS had developmental delay or intellectual disability (64%)
Nationwide newborn screening will help us to further refine these numbers.
3. What are the currently identified Fragile X-associated Disorders?
Fragile X syndrome (FXS): The most common inherited form of intellectual disabilities. FXS occurs in both genders, though boys tend to have more severe symptoms. It causes behavioral, developmental and language disabilities.
Fragile X-associated tremor/ataxia syndrome (FXTAS): An adult onset (over 50) progressive neurological condition (like a combination of Parkinson’s and Alzheimer’s) causing tremors, balance and memory problems, primarily in male carriers, which can, in some instances be life ending.
Fragile X-associated primary ovarian insufficiency: (FXPOI): A cause of infertility, early menopause and ovarian problems in women who are carriers.
Many more people with fragile X premutations (male and female) have noticeable symptoms however this spectrum of conditions is not currently described in the medical literature as a defined syndrome.
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4. How do you test for Fragile X?
A simple DNA blood test can conclusively determine if an individual has fragile X syndrome, or
is a Fragile X carrier.
5. What is the relationship between Fragile X and Autism?
Fragile X is one of the autisms. The perceived differences between FX and Autism are a function
of artificial constructs not of scientific reality. Autism and Fragile X share a relationship, the
science that is important to FX is important to Autism and the science that is important to Autism
is important to FX
The mutation that causes FXS also causes autism. FX is the most common known genetic cause of autism. The study of FX provides a unique opportunity to find the cause of and treatments for autism.
Studies published in 2012 have also established that the FX protein regulates or controls up to one half of the many hundreds of other genes believed to be responsible for causing autism.
Groundbreaking drugs to treat FXS (based on research funded by NIH) are also in clinical trials for individuals with autism)
Up to two-thirds of all children diagnosed with FXS also have autism, an autism spectrum disorder or behaviors associated with autism.
6. What is the relationship between Fragile X and other medical conditions?
Research into Fragile X will help shed light on other conditions such as Alzheimer's, Parkinson’s,
reproductive problems in women and other medical conditions that may have a genetic basis.
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Likely Questions from Congressional Staff 1. Who should I contact to express my support for Fragile X requests?
HOUSE OF REPRESENTATIVES
Congressman Gregg Harper (R – MS)
o Staff contact: Scot [email protected]
Congressman Eliot Engel (D-NY)
o Staff contact: Heidi Ross [email protected]
UNITED STATES SENATE
Senator Debbie Stabenow (D – MI)
o Staff contact: Kim Corbin [email protected]
Senator Johnny Isakson (R – GA)
o Staff contact: Chance Phiniezy [email protected]
William Dent [email protected]
2. How much money is the federal government currently devoting to Fragile X at the NIH and CDC?
CDC: ~ $1.7 million in FY 2013
NIH: ~ $28 - $29 million a year across all institutes over the last several years
DOD: ~$3 million a year beginning in 2011
3. How many people in my state are impacted by a Fragile X
Nearly 1 in 200
Take the total population of your State and divide by 200. That will give you an estimate of
the number of individuals who have or could develop a FXD.
4. Who Should I Contact to Learn More about the ABLE Act?
Barbara Riley, Comm Dir, Rep Crenshaw - 202-225-2501; [email protected]
April Mellody, Comm Dir, Sen Casey - 202-224-6324; [email protected]
John Rizzo, Press Sec, Sen Casey - [email protected]
5. If I have further questions regarding these requests or the National Fragile X Foundation's priorities who should I contact?
Jeffrey Cohen Interim Executive Director, NFXF 313 806 1190 [email protected]
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(Draft) Dear Colleague Letters from Senate and House Champions
Support Fragile X Research
March 5, 2014
(Senate) (Senate and House letters are in your Leave-Behind Folders)
Dear Colleagues:
As Members of Congress interested in children’s health and the promise of medical research, we invite you
to join us in expressing support for sustained federal investments in biomedical research and public health
initiatives focused on the treatment of Fragile X and its related conditions.
Fragile X-associated Disorders (FXD) are medical and developmental disorders that result in
behavioral, developmental and cognitive disabilities across a person’s lifespan. Fragile X syndrome
results from a single-gene mutation and is the most common, known inherited form of intellectual
disabilities and cause of autism. Recent research has shown that the Fragile X protein regulates
(controls) nearly one half of the genes suspected of causing autism! Fragile X mutations are also
linked to reproductive problems and early menopause in women (FXPOI) and a
Parkinson/Alzheimer-like condition in FX carriers (FXTAS). Over 100,000 Americans have fragile
X syndrome and nearly 1.5 million carry a Fragile X mutation and either have, or are at risk for
developing a Fragile X-associated Disorder.
Previous Congressional support of the important work underway at the NIH, CDC and DOD has made a
considerable impact on the lives of all Americans impacted. The CDC has recognized the significant public
health implications of Fragile X and has provided resources to ensure the continued growth and evolution of
the Fragile X Clinical & Research Consortium. Tremendous outcomes have already been produced with
relatively small amounts of money over the past few years. Current NIH supported clinical trials by
multiple drug companies are pointing the way to targeted treatments which may ameliorate many of the
core symptoms of fragile X syndrome and autism too!
To ensure that this exciting research leading to targeted treatments and unlocking the secrets of autism
continues, it is important that we maintain the federal initiatives that have made this possible and urge
federal agencies funding FX research and programming to more efficiently allocate available funding. This
includes translational research at NIH, the CDC’s national Fragile X public health program and Fragile X
Clinical & Research Consortium, and the Department of Defense Peer Reviewed Medical Research Program
(PRMRP).
Please consider joining us in expressing support for FXD by adding your name to the attached letters. If you
are interested in signing on, please contact either Francie Pastor in Senator Johnny Isakson’s office
([email protected]) or Kim Corbin in Senator Debbie Stabenow’s office
([email protected] ). The deadline to sign on to the letters is March , 2014.
Sincerely,
Johnny Isakson Debbie Stabenow
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(Draft) Sign-on Letters of Support to Appropriators from Senate and House
Champions (Draft 1-Senate L-HHS) The Honorable Tom Harkin The Honorable Jerry Moran
Chairman Ranking Member
Appropriations Subcommittee on Labor Appropriations Subcommittee on Labor
Health and Human Services, and Education Health and Human Service, and Education
131 Dirksen Senate Office Bldg. 354 Russell Senate Office Bldg.
Washington, D.C. 20510 Washington, D.C. 20510
Dear Chairman Harkin and Ranking Member Moran:
As Members of Congress committed to improving the health of children and adults living with
intellectual disabilities in the United States, we respectfully request your continued commitment to
sustaining federal investments in biomedical research and public health initiatives focused on the
treatment and cure of Fragile X and its related conditions.
Fragile X-associated Disorders (FXD) are medical and developmental disorders that result in behavioral,
developmental and cognitive disabilities across a person’s lifespan. Fragile X syndrome results from a
single-gene mutation and is the most common, known inherited form of intellectual disabilities and
cause of autism. Recent research has shown that the Fragile X protein regulates (controls) nearly one
half of the genes suspected of causing autism! Fragile X mutations are also linked to reproductive
problems and early menopause in women (FXPOI) and a Parkinson/Alzheimer-like condition in FX
carriers (FXTAS). Over 100,000 Americans have fragile X syndrome and nearly 1.5 million Americans
carry a Fragile X mutation and either have, or are at risk for developing, a Fragile X-associated Disorder.
The Committee’s previous support of the important work underway at the National Institutes of Health (NIH) and
Centers for Disease Control & Prevention (CDC) has had a considerable impact on the lives of all Americans
impacted. The CDC has recognized the significant public health implications of Fragile X and has provided
resources to ensure the continued growth and evolution of the Fragile X Clinical & Research Consortium.
Tremendous outcomes have already been produced with relatively small amounts of money over the past few
years. Current NIH supported clinical trials by multiple drug companies are pointing the way to targeted
treatments which may ameliorate many of the core symptoms of fragile X syndrome and autism too!
To ensure the rapid translation of ongoing research into targeted treatments that will be realistically
possible in the near future, we must continue these federal investments in the FY15 Labor, Health and
Human Services, and Education Appropriations bill. Specifically, we respectfully request your support
for directives to:
Create greater efficiency and synergy among the FX and autism research tracks to accelerate translational
research toward a better understanding of both conditions and shorten the time to bring effective
treatments for both conditions to market.
Maintain dedicated support for CDC’s national Fragile X public health program and continue to support
the Fragile X Clinical & Research Consortium.
While we understand the challenges the Committee faces in prioritizing requests, in light of:
-the significant impact Fragile X has on families and communities across the country
-the great potential for effective treatments that is within reach and -recent research has shown that the fragile x protein regulates (controls) nearly half of the approximately
300 genes suspected of causing autism,
We believe support for Fragile X research and public health activities is imperative.
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We thank you for your consideration
Sincerely,
_________________________ _________________________
Debbie Stabenow Johnny Isakson
Note: First, our Senate and House champions send a letter to their colleagues (a Dear Colleague letter) asking them to support FX by signing-on to a second letter to Appropriators which asks the Appropriations Committee to accept our proposed appropriations language in next year’s budget. Then, once signed, this second letter is sent to the Appropriations Committee for their consideration before next year’s budget is finalized. So in your meetings when you ask your Members to “sign the letter” or “sign on” you’re asking them to put their names on this second letter to Appropriations. These letters are reproduced here for your review. These letters are also in the Leave Behind Packets (one for each meeting) that you will receive at the end of the training day and that you will leave with each Member or Staffer you meet with. The draft above is a Senate letter. The same letter will circulate in the House from our house champions Greg Harper and Eliot Engel.
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Section VII: What to Do When You Go Home Meeting Feedback:
o If you have not already provided Meeting Feedback for each of your meetings on the
Soapbox app, please do so as soon as you get home on the app or online at
www.sboxapp.com
o Please be sure to note any request for follow-up information by Members/Staffers
Send a Thank You Letter:
Note: After Advocacy Day you will receive an E-Action Alert with a link to a prepared thank you letter that you can personalize and email. You should have the business card of the staff member that was in your meeting. Ideally you should send thank you letters to the staffer and the Member. This can be typed or handwritten and can be mailed or emailed. Use the sample letter below or create your own but make it personal and stay on point. The letter below is to the Senator or Representative. For staff you need to adjust the letter accordingly.
Dear Representative/Senator _______________:
Thank you for meeting with me during my visit to Washington, D.C.. It was a pleasure to participate in the legislative process, and I appreciate your time and interest.
ADD IN YOUR PERSONAL STORY HERE (I have a 16 year old son living with fragile x syndrome etc. etc.)
On March 5, 2014, I visited your office as part of the 11th National Fragile X Foundation's Annual Advocacy Day. We sought your support for various public health and research priorities to help my family and others living with a Fragile X-associated Disorder.
Our main requests are to:
1. Sign the letter to Appropriators that is being circulated by our champions (Senators Stabenow abd Isakson and Congressman Harper and Engle) to support key federal programs focused on Fragile X-associated Disorders.
2. Sign the letter to the House Energy & Commerce Committee that is being circulated by our champions in the House (Harper/Engle) urging them to convene a hearing on the intersections between Fragile X and autism.
Each of these requests will have a profound impact on the Fragile X community, and will play a vital role in supporting families living with fragile X syndrome, FXTAS, and/or FXPOI. I also shared with your staff my request for your support for legislation that would greatly enrich the lives of individuals with Fragile X and their families. Specifically, I would ask that you consider co-sponsoring:
3. CoSponsor the Achieving a Better Life Experience Act (ABLE Act) S. 313; H.R. 647
If you have already co-sponsored this bill, I thank you for your support.
Again, thank you for taking time out of your busy schedule. On behalf of my family and the National Fragile X Foundation, I appreciate your listening to my concerns regarding Fragile X. I look forward to hearing from you soon on whether you were able to support my requests.
Sincerely,
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And send a personal note to the Member and the Staffer you met with:
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Handy One Page Reference
The Opening – Introductions!
The Asks: 1. Sign the letter to Appropriators that is being circulated by our champions (Stabenow/Isakson –
Senate and Harper/Engle – House) to support key federal programs focused on Fragile X-associated Disorders:
o Maintain dedicated support for the CDC’s national Fragile X public health program and continue to support the Fragile X Clinical & Research Consortium.
o Create greater efficiency and synergy among the FX and autism research tracks at NIH, CDC and DOD to accelerate translational research toward a better understanding of both conditions and shorten the time to bring effective treatments for both conditions to the market
o Support for the continued inclusion of Fragile X-associated Disorders among the list of eligible healthcare conditions for targeted biomedical research funding through the U.S. Department of Peer Reviewed Medical Research Program.
“Please sign our Champions Letter to Appropriators –– a copy is in the leave behind folder!”
2. Sign the letter to the House Energy & Commerce Committee that is being circulated by our champions in the House (Harper/Engle) urging them to convene a hearing on the intersections between Fragile X and autism. This will:
o ensure the Department of Health & Human Services (HHS), the National Institutes of Health (NIH), industry, and other key stakeholders are providing appropriate investments in these areas and believe a Congressional hearing in the House Energy & Commerce Committee focused on these intersections would yield significant attention to these important issues for people with Fragile X Associated Disorders and autism
“Please sign the letter to the House Energy and Commerce Committee supporting our request for a hearing on the intersections between Fragile X and autism.” Scott Malvaney in Congressman Gregg Harper’s office is the contact
o As an FYI, mention to Senate offices that we are pushing for a hearing on this topic to House Energy & Commerce Leadership.
3. Co-Sponsor pending legislation to help us pass the ABLE Act in the 113th Congress o The ABLE Act will have an immediate impact on the life of my (son/daughter/family
member/family) impacted by a Fragile X-associated Disorder and will allow me to begin saving for his/her future and help him/her reach the highest level of self-sufficiency and independence possible.
o Co-Sponsor – if the Member is already a cosponsor, thank them for their support of the bill and remind them that there over 400 Members of Congress also supporting this bill. If they are NOT a cosponsor, please ask them to cosponsor the bill and tell them why it’s important to our community.
o Sign onto the letter to the House/Senate leadership which requests that the ABLE Act be brought to the floor for a vote as soon as possible. *By March 7th HOUSE: Jennifer Debes in Rep. Crenshaw’s office ([email protected]) SENATE: Jennifer McCloskey in Sen. Casey’s office ([email protected])
“Please contact the offices of the ABLE Act’s champions and let them know that you will support and vote for passage of the bill in this Congress.” The Close – Thank you and follow up!
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Section VIII: Contact Info
CATEGORY KEY CONTACTS CONTACT INFO
NFXF DC Jeffrey Cohen, Interim
Executive Director, Director
Government Affairs and
Advocacy
313-806-1190
NFXF Board Leadership
Soapbox
Rep. Harper’s
Office
Jimi Grande, Chair Public Policy Committee
202-255-2810
Soapbox Help Desk Ask for Emily Hoff-she won’t be able to help with specific scheduling but if you need help with anything else call her
202-362 5910 202-225 5031 or [email protected]
NFXF Staff Jeffrey Cohen 313 806 1190
Linda Sorensen, Associate Director
925-283-1897
David Salomon, Communications Coordinator
925-385-8431
Your Mentor
Capitol Hill Operator
If you need to contact any congressional office for any reason, dial this number and ask to be transferred to any Senate or House office.
202-224-3121
Emergency Numbers for Capitol Hill
Emergency
911
Capitol Hill Police – Non Emergency
202-225-5151
Washington DC Police – Non Emergency
202-727-1010
HOTEL INFORMATION
Sheraton Four Points
(202) 289-7600
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METRO INFO METRO INFO
Closest to Capitol Hill – House Side
Capitol South Metro (Orange/Blue Lines) House Side
Closest to Capitol Hill – Senate Side
Union Station (Red Line)
Closest to Hotel (Sheraton) Online Metro Map PRINT IT OUT BEFORE YOU ARRIVE METRO APPS ARE AVAIL
McPherson Station-Orange and Blue http://www.wmata.com/rail/maps/map.cfm At itunes and Google Play-Search for DC Metro
Soapbox has a free Smartphone App for iPhone and Android. Search for: Soapbox Advocacy Day App and download it to your smartphone before you arrive. On Tuesday (training day) you will be provided with your personal code and will then be able to access your schedule (with last minute changes), team members, map of Capitol Hill, Member bio’s and the Soapbox Helpdesk from your phone.