Expert Interview
Discussing the second death of a patient who was treated with Elevidys the effect on future prescribing and uptake
Ticker(s): SRPTInstitution: Connecticut Children’s
- Pediatric neurologist and Division Head of Neurology at Connecticut Children’s
- Extensive experience in neuromuscular research and clinical trials, including work related to gene therapy and emerging treatments for Duchenne, SMA, and other muscular dystrophies, contributing to translational efforts aimed at improving outcomes for children with DMD and SMA
- Currently manages approximately 20 patients with Duchenne muscular dystrophy and 27 patients with spinal muscular atrophy, overseeing long-term neuromuscular care including motor function management, respiratory and cardiac coordination, and integration of emerging therapeutic options.
How do you think the recent death might affect perception around risk/reward in older, non-ambulatory patients?
Added By: wilson_adminDo you think the death will affect your prescribing patterns?
Added By: wilson_adminOverall, what is your feedback of Elevidys in terms of efficacy/safety so far?
Added By: wilson_adminAre You Interested In These Questions?
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