Expert Interview
Discussing the potential of Sarepta Therapeutics’ investigational gene therapy, SRP-9001 (delandistrogene moxeparvovec) for the treatment of DMD with a pediatric cardiologist.
Ticker(s): SRPTInstitution: University of Tennessee Methodist Hospital - Memphis
- Chair in Cardiology, Professor and Chief of Adult Cardiovascular Diseases, and Professor of Pediatric Cardiology at the University of Tennessee Methodist Hospital.
- Treats 30 patients with Duchenne Muscular dystrophy (DMD).
- Research focuses on gene discovery for cardiomyopathy and novel therapeutic strategies for heart failure; Clinical focus on heritable forms of cardiovascular disease such as cardiomyopathies, aortopathies, channelopathies, and other genetically triggered diseases.
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: sara_adminHow 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: sara_adminThere 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: sara_adminAre You Interested In These Questions?
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