Leading the Fight to treat and cure
Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

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News Flash

New FAQ on Clinical Trials

What Clinical Trials are open? How do they work? What are the risks? NTSAD realizes there will be many questions regarding clinical trials in general, as well as specific questions related to the trials for the diseases that impact families. Read Clinical Trial FAQ

 

Juvenile GM1 Trial Open

Gene Therapy trial now enrolling children ages 2-12 with type II GM1 gangliosidosis. The study is to determine if a gene therapy can help the body to make an enzyme. Read details at ClinicalTrials.gov

Axovant 1st Evidence of GM2 Stabilization

At the european society of gene and cell therapy 27th annual congress, Axovant presents first evidence of clinical stabilization in tay-sachs disease. Read Press Release

2019 Day of Hope Success!

Through the efforts of families and friends, over $60,000 was raised during the 2019 Day of Hope, bringing our overall Day of Hope total to over $500,000 since 2011!

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Over $140,000 Raised at 2019 Imagine & Believe

Together, with our friends, we honored Dr. Cynthia Tifft at the October 30, 2019 Imagine and Believe evening at the Sonesta Hotel in Cambridge. Dr. Tifft is Deputy Clinical Director of the National Human Genome Research  Institute/NIH. We imagined and believed and we raised over $140,000!

Read more ...

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