-
NTSAD Annual
Family Conference
April 11-14, 2019Click here for more information, registration, and to see our generous sponsors and Helping Hand Grant supporters! Read More -
Another step forward
for GM2 and GM1
gene therapy programs.Excited to have gene therapy programs in development for GM1, GM2 and Canavan diseases. Read the latest GM2 update from Axovant with promising results. Read More -
Calling all families!
Your experiences past and present are needed!Please take a moment to register with the Patient Insights Network (PIN) and help researchers! Read More
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