Sara’s Story
A Message from Sara’s Mother Hello Friends and Families We are an affected family from…
Florence’s Story
Florence has late infantile GM1. She was diagnosed when she was 2. She has real…
Axovant Intravenous AAV9 Gene Therapy 2019 Clinical Trial Announced!
GM1 Intravenous AAV9 Gene Therapy Clinical Trial to Begin in the First Half of 2019…