Charlene Son Rigby, President and Co-founder of the STXBP1 Foundation, interviews ENDD’s Mike Boland about STXBP1 therapeutic development updates from the annual meeting of the American Society for Gene and Cell Therapy.
From The Transmitter, by Lydia Denworth.
A handful of scientists are committed to advancing research on the autism-related genetic conditions their own children have.
In this piece, “Accelerating therapeutic development and clinical trial readiness for STXBP1 and SYNGAP1 disorders,” Nick, Mike, and Ben provide an overview of the disorders’ clinical features and the current understanding of pathogenic mechanisms and therapeutic development.