Expert Interview
Discussing the Phase III trial results of intrathecal onasemnogene abeparvovec (OAV101 IT), exploring its efficacy and safety in treating a broad patient population with spinal muscular atrophy
Ticker(s): NVSInstitution: 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
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