The CGTLive™ Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. It also contains up-to-date clinical news coverage in the field of gene and cell therapy for central nervous system disorders.
April 27th 2024
The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year.
FDA Revisits Need for Advisory Committee on Sarepta’s DMD Gene Therapy
March 18th 2023Ahead of the May 29, 2023, action date, the FDA is now seeking to hold an advisory committee meeting on Sarepta’s investigational agent SRP-9001 (also known as delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy.
Microdystrophin Proposed as a Surrogate End Point in DMD
March 16th 2023Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.
Jeffrey S. Chamberlain, PhD, on Regulating DMD Gene Therapy Clinical Trials
March 9th 2023The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed working with the FDA toward a first disease-modifying therapy approval in Duchenne.