Expert Interview
Discussing the potential of Sarepta Therapeutics’ investigational gene therapy, SRP-9001 (delandistrogene moxeparvovec) for the treatment of DMD with a pediatric neurologist.
Ticker(s): SRPTInstitution: Duke University
- Co-Director of Duke Children's Neuromuscular Program & Professor of Neurology and Pediatrics at Duke.
- Treats hundreds of neuromuscular patients, including 150 Duchenne Muscular Dystrophy patients and 60 Spinal Muscular Atrophy patients
- Actively involved in clinical research and trials with a focus on neuromuscular diseases; PI on multiple DMD studies including an Exon 53 skipping specific study.
Roughly how many patients do you manage with DMD tumors?
Added By: sara_adminCan you talk about the clinical potential of delandistrogene moxeparvovec in patients with DMD?
Added By: sara_adminOn a scale from 1-10 (10 being extremely excited) where would you rate your level of excitement for Nirogacestat?
Added By: sara_adminWhat risk-benefit profile do you expect for SRP-9001 in terms of functional endpoints and biological endpoints (micro-dystrophin level)?
Added By: user717c8673How do you compare another recent NDA filing of vamorolone for DMD? Do you see them competing for the future standard of care, or used in combination?
Added By: user717c8673There are many clinical metrics to measure motor function as clinical endpoints, NSAA, TTSTAND, 6MWT, RRTW, etc.. Do you see them being used in combination or some are preferred over others? Do you see them used differently in different subgroups (e.g., by different age groups, NSAA may plateau after 9 yo)
Added By: user717c8673Are You Interested In These Questions?
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