It was a pleasure to meet with Jerry Mendell (Nationwide Children’s Hospital, Columbus, OH, USA) to discuss his recent work in the gene-delivery clinical trial of rAAVrh74.MHCK7.micro-dystrophin (SRP-9001) for Duchenne Muscular Dystrophy.
The abstract entitled: ‘A Multicenter Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy’ was presented at the American Academy of Neurology Virtual Annual Meeting 2021.
Questions:
- What preclinical and clinical evidence supports the investigation of intravenous rAAVrh74.MHCK7.micro-dystrophin (SRP-9001) for patients with DMD? (0:05)
- What was the design and eligibility criteria of the clinical study you are presenting? (1:06)
- What have the initial efficacy and safety data taught us? (2:37)
- What will be the next step in the clinical development of SRP-9001? (3:42)
Disclosures: Jerry Mendell has received honorarium, consulted for, and worked as a Principal Investigator for Novartis and Sarepta therapeutics; and has received honorarium, consulted for and is part of the Scientific Advisory Board for Vertex Pharma.
Support: Interview and filming supported by Touch Medical Media.
Filmed as a highlight of AAN Annual Virtual Meeting 2021.
