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Goodbye March & Hello April. 

RaiseRight Webinar

Join our upcoming webinar, Tuesday April 16th to discover how your everyday purchases can effortlessly contribute to Cure GM1. Learn the simplicity of using gift cards from over 750 major retailers to support our cause. Imagine turning your grocery shopping, gas fill-ups, and coffee runs into significant donations. Register now to find out how every swipe can bring us closer to a cure. Can’t join, see our RaiseRight instructions by clicking the Learn More.

Cure GM1 RaiseRight Webinar
Cure GM1 Virtual Annual Conference

2024 Virtual Annual Conference

Cure GM1 New Team Member

David Weinstein, MD, MMSc
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Say Hello

Cure GM1 New Team Member

Anita Hernandez
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Cure GM1 Proclamation for Violet

A Proclamation for Violet & the GM1 Community

Cure GM1 T-Shirts

Votes are In

Cure GM1 Surpasses $5M in funds raised!

Years ago, we had not a single cent dedicated to our cause.  Thanks to the incredible support of our global GM1 community, we can now plan for new research and have funds to be able to seed meaningful research and to attract greater investment.  Cure GM1 has several projects in the contracting phase which will be announced as soon as possible. While there is still much work to do and funds are continually a challenge due to the high price of scientific research and drug development, we continue to push forward.

Cure GM1

Creating Hope Reauthorization Act Introduced

 On February 15, the Creating Hope Reauthorization Act was introduced in the House by Representatives McCaul, Bilirakis, Burgess, Barragan, Eshoo, and Trahan. This legislation would reauthorize the Food and Drug Administration (FDA)’s Rare Pediatric Priority Review Voucher program, which incentivizes pharmaceutical companies to develop new drugs for rare pediatric diseases. To learn more on the Creating Hope Reauthorization Act, Click Here. To learn more about the PRV program, Click Here.

Cure GM1 BioTech News

MLD Gene Therapy Approval

Can MLD serve as an example for GM1?  Published research supports that the very same lentiviral gene therapy approach could be applied to GM1.  What is lacking is an actual clinical trial and sponsor. This gene therapy approval for  MLD is a hopeful example for lysosomal diseases, leukodystrophies and rare diseases overall.  However, the reality is that this approval in the US and the overall process was lengthy with the first patients being treated 12 years ago.  And this therapy was approved in Europe earlier. Advocacy is critical in continuing to push for progress in all rare diseases and for GM1. Read More.

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy. “In March the U.S. Food and Drug Administration approved Lenmeldy, the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).”  Read More.

Cure GM1 BioTech News

Azafaros completes Phase 2 trial in GM2 and NPC.  Phase 3 to include GM1, GM2, and NPC Phase 3

Cure GM1 BioTech News

RAINBOW Clinical Trial

Roberto Giugliani, MD, PhD, discusses the RAINBOW clinical trial testing the efficacy and safety of nizubaglustat for Niemann-Pick type C and GM2 gangliosidosis. Learn More.

Purple-petaled Glower Besode Pen

Mother’s Day Around The Corner

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