Expert Interview
Delving into the topline data from the Phase 2/3 DEVOTE study of higher dose Spinraza (nusinersen) in patients with SMA
Ticker(s): BIIBInstitution: Rare Disease Research - NC (formerly Duke University)
- Medical director and principal investigator for Rare Disease Research - NC and Former Co-Director of Duke Children's Neuromuscular Program & Professor of Neurology and Pediatrics at Duke.
- Treated hundreds of neuromuscular patients, including 150 DMD patients of all ages in 2023.
- Actively involved in clinical research and many trials with a focus on pediatric neuromuscular diseases; PI on multiple DMD studies including an Exon 53 skipping specific study.
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