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Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder of upper and lower motor neurons that results in progressive motor impairment. ALS is the most common disease of motor neurons with an annual incidence of approximately 1.7–2.5 per 100,000 people. It is a terminal condition with a typical life expectancy of 2–5 years from symptom onset. […]

Jerry Mendell, AAN 2021: Gene Therapies in the Treatment of Patients with Duchenne Muscular Dystrophy

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Published Online: Apr 27th 2021

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:

  1. What are the limitations of current treatment options for Duchenne muscular dystrophy (DMD)? (0:05)
  2. 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.

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