The Cure GM1 Catalyst
NEWSLETTER
Discover how Cure GM1 is making an impact. We are thrilled to welcome two new advisors to our team and to join the Compassionate Friends global candle-lighting ceremony honoring children gone too soon. Plus, get updates on critical legislative advocacy, our engagement with the broader rare disease community, Giving Tuesday successes, and heartwarming holiday stories.
FUNDRAISING
ADVOCACY
COMMUNITY & ENGAGEMENT
ANGELVERSARIES & BIRTHDAYS
Thank you so much for being part of our Giving Tuesday campaign.
FUNDRAISING
Thanks to your generous participation, we received $39,084 in donations including matching funds. Your support means everything to us and the families affected by GM1. This funding is crucial for:
Additional funding can help to expand our xCures natural history pilot to include more participants, providing invaluable data. Our advocacy efforts continue to keep drug development moving forward. We continue to work to launch new initiatives to explore potential treatments, offering hope to children and their families.
Because of you, we’re one step closer to these breakthroughs. We cannot thank you enough for standing with us in this critical effort. Together, we’re not just raising funds – we’re creating hope and changing the future of GM1.
Let’s finish the year strong!
FUNDRAISING
As part of our amazing community, you know the impact we can make together. With your support, we can keep advancing research, advocacy, and hope for every family affected by GM1.
As you’re wrapping up your shopping list this season, don’t forget a year-end gift to the Cure GM1 Foundation. Plus, your year-end gift offers potential tax benefits, it’s the perfect time to give! Let’s finish 2024 strong together. Will you help us reach the finish line?
Navigating the Holidays with a Special Needs Child
Advocacy
The holiday season is full of joy and togetherness, but it can also bring unique challenges for families with special needs children. These seven practical tips will help you create a smoother, more enjoyable holiday experience for your child and family. From planning to creating sensory-friendly environments, these strategies can make a world of difference. Read the full article for detailed guidance and helpful insights.
Rare Pediatric Disease Priority Review Voucher Program Status
ADVOCACY
The Rare Pediatric Disease Priority Review Voucher Program has still not been renewed past December 20, 2024. Stay updated on the latest developments. Read more >
Meanwhile, H.R. 7384, the “Creating Hope Reauthorization Act,” would amend the date by which the drug must be designated as a rare pediatric disease drug to September 30, 2028, and the date by which the application must be approved to September 30, 2030. This bill was introduced in the House and referred to the Energy and Commerce Committee’s Subcommittee on Health.
If the rare pediatric disease PRV program is not reauthorized, eligibility to receive a voucher will be limited to products that receive rare pediatric disease designation by December 20, 2024, and are approved by September 30, 2026.
Action Alert: Contact your Senator today to ensure the “Creating Hope Reauthorization Act” passes before December 20th, 2024!
Share Your Holiday Spirit with the GM1 Community
COMMUNITY & ENGAGEMENT
As the year wraps up, we’re spreading awareness and recognizing our global community, and we’d love for you to be part of it! Share a high-resolution holiday photo of your family or children, along with a meaningful holiday quote. Please note that all submissions will be shared publicly within our community. Submit your holiday quote using this form: Holiday Quote Form.
Sweet Iris Day Was December 3rd
COMMUNITY & ENGAGEMENT
December 3rd was Sweet Iris Day, a day dedicated to raising awareness for GM1 gangliosidosis. This special day was proclaimed by our mayor in honor of Iris, who, at just 9 years old, inspired our community with her strength and spirit. Join us as we shine a light on GM1 awareness and advocacy.
Welcoming Chris Phillips and Paul Fitzpatrick
to the Cure GM1 Advisory Team
COMMUNITY & ENGAGEMENT
We are pleased to welcome Chris Phillips and Paul Fitzpatrick as new advisors to Cure GM1. Their expertise and commitment will strengthen our efforts to support the GM1 community and drive meaningful progress.
Paul Fitzpatrick, PhD
Scientific Advisor
Paul Fitzpatrick has nearly 30 years of biotechnology leadership experience including an impactful 20-year career at BioMarin, where he led the Biologics and Production Group, including scientific and operational responsibility for research stage biologics (ERTs) and AAV production, as well as early stage process analytics and structural, cellular, and protein biology fundamental research. Since then, Paul has worked at Zogenix, Inc. and Chameleon, Inc. doing further CMC and research work in AAV gene therapy.
Chris Phillips
CMC Advisor
Decades of CMC experience in the biotech/pharmaceutical industry spanning R&D, cGMP plant manufacturing operations and third party management. Graduate degree in plant biochemistry. Headed CMC for FDA approvals for 2 BLAs (Pegaspargase® (pediatric leukemia) and Revcovi® (severe combined immunodeficiency disorder)) and 2 NDAs (Periostat® doxycycline tablets (periodontal disease) and Eskata® 40% hydrogen peroxide applicators (seborrheic keratosis).
Read More>
Christine Represents Cure GM1 at the Ultragenyx Rare Disease Bootcamp
COMMUNITY & ENGAGEMENT
Christine attended the Ultragenyx Rare Disease Bootcamp on November 12th and 13th, connecting with a dedicated community of advocates working to develop new treatments for rare diseases. While Cure GM1 has been tirelessly fighting this battle for years, staying connected with advancements in technology and expanding our network is essential to driving progress and hope for our families.
Read More>
Join the Worldwide Candle Lighting Ceremony
COMMUNITY & ENGAGEMENT
Worldwide Candle Lighting Ceremony On December 8, 2024, at 7:00 pm local time, candles will be lit around the world to honor the memory of children gone too soon. The annual Worldwide Candle Lighting (WCL), organized by The Compassionate Friends, is now the largest mass candle-lighting event globally.
Love Letters to Our Angels
COMMUNITY & ENGAGEMENT
Purchase Prints and Cards to Support Cure GM1 year-round with Minted.com!
fundraising
Use the code FUNDRAISECGM1 at checkout to receive a special discount on your purchases. Plus, 15% of your purchase will be donated to charity. It’s a simple way to give back while shopping for beautiful, high-quality products. Every purchase helps make a difference for those affected by GM1 gangliosidosis.
Visit Minted.Com to support Cure GM1
Winter Flowers
FUNDRAISING
This holiday season, let the beauty of fresh flowers brighten your celebrations and warm the hearts of your loved ones. Whether adorning your table, gifting a friend, or bringing a festive touch to your home, our handcrafted holiday arrangements are designed to spread joy and a portion of your purchase will support GM1 research.
Order Here>
RaiseRight
FUNDRAISING
RaiseRight sells gift cards you can use to pay for everyday and special expenses. A percentage of the gift card goes to Cure GM1 as a donation, directly from the brand at no extra cost to you – as much as 17%! Get inspired with this interactive flipbook. From holiday prep to gifts for everyone on your list, RaiseRight is the easiest way to earn for your organization.
Winter See’s Candies
FUNDRAISING
Treat yourself or a loved one to delicious See’s Candies chocolates this winter, and a portion of your purchase will support GM1 research. Every box brings us closer to a cure while spreading joy and sweetness to those you care about. Order now and make your sweet treats count for a great cause!
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