FEBRUARY 2025 ADVOCACY NEWSLETTER | |
FY24 CDMRP Awards Announced! | |
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FARA and NAF are thrilled to announce that four Hereditary Ataxia grants, totaling over $12.25 million, have been recommended for funding through the Fiscal Year 2024 Congressionally Directed Medical Research Programs (CDMRP)! Ataxia was added to this program three years ago and with the addition of these awards, there have been seventeen grants recommended for funding, totaling over $36 million.
This funding would not have been possible without our advocates! Each year, during the budget cycle, the US Congress selects conditions eligible for this grant program. The process to get on the list starts when a Congressional member receives an “appropriations request” from a constituent. Basically, someone needs to ask for it! Each member then reviews the appropriations requests they receive and decides which ones to forward on to the appropriations committee for further consideration. Members of our community throughout the US, working in collaboration with FARA and NAF, not only made the initial requests but also followed up through meetings, emails and calls to explain the relevance of Ataxia to the goals of the CDMRP program as well as the potential benefits of investing in Ataxia research. The FY25 budget is currently under review by Congress. Appropriations requests for FY26 budget just opened and will continue throughout the spring. The CDMRP has been an extraordinary boost to ataxia research, and working together, it will continue to be a viable funding source for Ataxia research.
The CDMRP initiative is certainly a wonderful example of the power of our collective voice, but our community impact has been much broader. Together, we have raised awareness of ataxia, supported funding for research agencies, and helped pass legislation and policies to expedite research. With the transition happening in DC, your voice matters more than ever. Sharing your life experiences is the key to lawmakers understanding the challenges and unmet medical need our community faces, which will pave the road to legislative actions that can expedite treatments.
The timing could not be better! On February 28, the world will recognize Rare Disease Day. Below is an amazing list of events that you can participate in to help raise awareness of rare disease in general, and ataxia specifically. Pick one that works for you and join FARA and NAF in advocating to cure Ataxia.
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While registration is now closed for this in-person DC event, there are still ways that you can be involved from home! The EveryLife Foundation will provide virtual viewing of several events listed below here. This is a great opportunity to learn more about the issues impacting the rare disease community!
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Rare Disease Week on Capitol Hill | |
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Monday, February 24: Documentary screening (5-8 PM ET)
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Tuesday, February 25: Legislative Conference (9 AM-5 PM ET)
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Wednesday, February 26: Rare Disease Congressional Caucus Briefing (9-10 AM ET)
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The FDA and NIH will be holding a joint event for Rare Disease Day from Thursday, February 27 and Friday, February 28. This event is free to attend and has both an in-person and virtual option, allowing you to attend even if you can’t make it to DC! This event will feature patient stories, highlight recent rare disease research supported by the FDA and NIH, and provide additional updates to the rare disease community. | |
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In addition to the action happening in DC, there are also a number of other events happening across the country, including:
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Rare Disease Symposium, February 20, 2025, at Washington University Medical Center in St. Louis, Missouri, or via Zoom
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Oklahoma Rare Disease Day, February 20, 2025, at the Oklahoma State Capitol in Oklahoma City
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The Boston Globe’s Rare Disease Summit, February 25, 2025, at Big Night Live in Boston, Massachusetts
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CHOC & UCI Rare Disease Symposium & Family Conference, February 26, 2025, and February 28, 2025, at the Beckman Center in Irvine, California
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Rare Disease Day 2025 at the University of Minnesota, March 6, 2025, at the McNamara Alumni Center in Minneapolis, Minnesota
Check out NORD’s interactive page listing local Rare Disease Day events here to see if there are any happening near you!
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Make Your Voice Heard in New York This Rare Disease Day! | |
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Recently, a bill was introduced that would create a permanent Rare Disease Advisory Council (RDAC) in New York (S1287-A (Persaud)/A1296-A (Paulin). RDACs address the needs of rare patients and families by giving stakeholders an opportunity to make recommendations to state leaders on critical issues including the need for increased awareness, diagnostic tools and access to affordable treatments and cures. You can help get this legislation across the finish line by taking action here.
You can also join NORD in Albany for Rare Disease Day on February 25 from 9 am to 4 pm at the New York State Capitol Building. Advocates will be urging the passage of the RDAC bills and thanking New York lawmakers for the recent passage of the Rare Disease Day resolution, which proclaims February 28, 2025 as Rare Disease Day in the State of New York. You can register here.
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Rare Disease Activities from Home! | |
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If you cannot travel, there are still plenty of ways that you can get involved at home as you can see from the list below! Most of all, it only takes a few minutes to send an email to your lawmakers and your story might be the one that impacts the action they take. Remember, our asks — to support medical research and access to diagnostics, care and treatment — are bipartisan issues because ataxia affects all types of individuals across the country.
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Contact Your Legislators!
Contacting your elected officials and sharing your story is incredibly impactful even if shared via email or phone. First, look up your legislators here by entering your home address. Then, contact them through their websites. Here is a template to use when reaching out to your legislator:
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Hello Rep./Sen. [Insert name of Representative/Senator],
My name is [YOUR NAME], and I am your constituent from [CITY, STATE]. I am reaching out to you today to share my story with rare disease and encourage you to take action to support the needs of the rare community. I have a rare disease called [insert your type of ataxia], which is one of over 70+ forms of heredity Ataxia. Ataxia is a rare degenerative neuromuscular disorder that affects my mobility, coordination of movement, speech, and overall health.
[You can insert more of your personal story here to help educate your legislators!]
There are over 10,000 known rare diseases, of which 95% have no approved treatments. Hereditary ataxias are no exception. As of now, there are no treatments addressing the root cause of the disorder, and the treatments available only help to manage symptoms and/or slow progression. This is why adequate funding for agencies like the NIH and FDA is so crucial. Funding these agencies helps accelerate potential treatments and gives patients and families hope. I respectfully request that you do all that you can to pass the FY25 budget by the March 14 deadline and support lifting the NIH 15% cap on indirect costs in grants. Agencies cannot recruit or retain the best doctors and scientists when funding is not robust or timely.
I also ask that you work to reinstate the Rare Pediatric Disease Priority Review Voucher (PRV) Program. This program serves as a crucial incentive for drug development for pediatric-onset rare diseases, including the two types of childhood-onset Ataxia. With over 70% of rare diseases presenting in childhood and 30% of those children dying before the age of five, we need more incentives for pediatric drug development, not less. Moreover, the program presents no cost to taxpayers, making it a budget-neutral program with monumental impact.
Thank you for your time and consideration, and please don’t hesitate to reach out with any additional questions.
Best,
[YOUR NAME]
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NORD is running the following Rare Disease Day programs. Get creative! Have fun!
Raise Awareness and Show Your Stripes®
Show your support for Rare Disease Day and raise awareness for rare disease by decorating your home, neighborhood, school, or town with zebra stripes. Click here for more information on how you can Show Your Stripes® this Rare Disease Day.
If you are interested in free promotional materials and other Rare Disease Day activities, you can find those here.
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Light Up for Rare on Rare Disease Day®
Light Up for Rare® is an easy way to get involved in rare disease day in your community. Typically, you will need to get in touch with the landmark and may have to fill out a request form. It’s that easy to raise awareness for rare disease!
Global Chain of Lights
In an effort of global solidarity, you are invited to light or decorate your home with the Rare Disease Day colours at 7 PM your local time on 28 February 2025.
You can use garlands, social media filters, candles, disco lamps, colorful decorations… Let your creativity shine! If you choose to participate, take a picture of yourself with the illuminations or decorations you chose and post it on your social media using a “Global Chain of Lights” filter and #rarediseaseday.
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Raise Awareness on Social Media
There are so many ways to raise awareness about rare disease on social media! This could include:
- Sharing your rare disease story
- Posting a selfie in stripes and encouraging your friends and family to do the same
- Repost infographics and photos from other members of the rare disease community
- Or making other posts about rare disease and Ataxia!
If you intend to raise awareness on social media, make sure to use #rarediseaseday in your post! You can find more information and templates for posts in this social media toolkit!
For more information on how you can get involved on Rare Disease Day and additional resources to help you get started, take a look at NORD’s Rare Disease Day Community Playbook
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