Expert InterviewNVS
Slingshot members are talking to an expert! The topic is:
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
Executed on:May 8, 2025 2:00 PM EDT
Call Duration:30 minutes
Expert Profile
ExpertBlinded Expert
Institution: 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 PittsburghManages 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.
Interview Goal
On this call, we will discuss the perceived efficacy, safety, and real-world applicability of intrathecal onasemnogene abeparvovec (OAV101 IT) for treating spinal muscular atrophy, in light of recent Phase III trial data. We aim to understand how these findings may influence treatment decisions and integration into current SMA management protocols.