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
- Clinical and research interests include spinal muscular atrophy, muscular dystrophies, Charcot-Marie-Tooth disease, and genetic treatments for neurological conditions.
- Manages 27 patients with SMA and can speak to the anti myostatin inhibition approaches for SMA including Scholar Rock's apitegromab, Biohaven's taldefgrobep Alfa and Roche's GYM329
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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