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.
- What are the limitations of current treatment options for Duchenne muscular dystrophy (DMD)? (0:05)
- Could you tell us a little about the potential of gene therapies in the treatment of patients with DMD and the challenges associated with its use? (1:24)
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.
Share this Video
Related Videos In Neuromuscular Diseases
Teresa Coelho, WCN 2021: New Therapies in Hereditary ATTR Amyloidosis – Tafamidis, Patisiran, Inotersen and Future Therapies
touchNEUROLOGY met with Dr Teresa Coelho (Unidade Clinica de Paramiloidose, Hospital de Santo Antonio, Porto, Portugal) to discuss new therapies for the treatment of hereditary ATTR amyloidosis. Hereditary ATTR amyloidosis is a progressive and fatal disorder caused by variant transthyretin deposition as amyloid. The interview discusses an overview of tafamidis, patisiran, and inotersen, and their […]
Giuseppe Lauria Pinter, WCN 2021: The Unfolded Protein Response in ALS: Results of a Phase 2 Trial
touchNEUROLOGY met up with Prof. Giuseppe Lauria Pinter (University of Milan, Milan, Italy) to discuss the efficacy and outcome measures of a phase 2 study investigating guanabenz for the treatment of amyotrophic lateral sclerosis. The abstract entitled: ‘The unfolded protein response in Amyotrophic lateral sclerosis: Results of a Phase 2 Trial’ was presented at the […]
Renato Mantegazza, EAN 2021: Challenges in the Treatment of Myasthenia Gravis
touchNEUROLOGY met with Renato Mantegazza (Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Milan, Italy) to discuss the challenges that arise in treating patients with myasthenia gravis. The abstract entitled: ‘Safety and Tolerability of Efgartigimod in Patients with Generalized Myasthenia Gravis: Phase 3 ADAPT Study Results’ was presented at the 7th […]
Journal articles and more to your inbox
Get the latest clinical insights from touchNEUROLOGYSign me up!