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.

Executed on:Jun 20, 2024 2:00 PM EDT

Call Duration:30 minutes

Expert Profile

ExpertBlinded Expert

Institution: 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.

Interview Goal

On this call we will be discussing the new clinical data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD, and the potential of both candidates for their respective indication.

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