Expert Interview
Discussing the data from the Phase 1/2 ACHIEVE trial of DYNE-101 in patients with DM1 and the ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with DMD.
Ticker(s): DYNInstitution: Duke University
- Former Co-Director of Duke Children's Neuromuscular Program & Professor of Neurology and Pediatrics at Duke, recently made a career change and is now exclusively focused on clinical trials.
- 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 pedicatric neuromuscular diseases; PI on multiple DMD studies including an Exon 53 skipping specific study.
Roughly how many patients with DMD?
Roughly how many patients with DM1?
Due to your experience in clinical trials, can you talk a bit about the trial design for both of these studies and what endpoints you would want to see in future Phase 3 studies?
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