Expert Interview
Discussing the Phase III trial results of Novartis' intrathecal onasemnogene abeparvovec (OAV101 IT) in treating patients aged 2 to under 18 years with spinal muscular atrophy
Ticker(s): NVSInstitution: UPMC Children's Hospital of Pittsburgh
- Pediatric neuromuscular neurologist at the University of Pittsburgh and the Director, Pediatric Neuromuscular Program, UPMC Children's Hospital of Pittsburgh
Manages 35 patients with Spinal Muscular Atrophy - Collaborates on multiple multi-center research studies in the field of pediatric neuromuscular disease
- Research interests include Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, and neuromuscular disease in children including outcome measures, new therapeutic interventions and genetic therapies.
Roughly how many patients with SMA do you manage monthly?
Added By: dami_adminHow do you see OAV101 IT fitting into the current treatment landscape for SMA, especially in comparison to other available therapies like nusinersen or risdiplam?
Added By: dami_adminWhat is your initial impression of the Phase III trial results for intrathecal onasemnogene abeparvovec (OAV101 IT), particularly regarding its efficacy and safety in type 2 SMA patients?
Added By: dami_adminAre You Interested In These Questions?
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