Duchenne News

August 11, 2022
On August 10, 2022, Dyne Therapeutics announced the publication of Duchenne muscular dystrophy preclinical data: Enhanced Exon Skipping and Prolonged Dystrophin Restoration Achieved by TfR1-Targeted Delivery of Antisense Oligonucleotide Using FORCE Conjugation in Mdx Mice.
August 1, 2022
On July 29, 2022, Sarepta Therapeutics shared news regarding SRP-9001.
July 14, 2022
On July 12, 2022, Edgewise Therapeutics shared a press release announcing the opening of the CANYON Phase 2 clinical trial of their investigational compound, EDG-5506, in individuals with Becker muscular dystrophy.
July 6, 2022
On July 6, 2022, Sarepta Therapeutics shared new functional data across multiple studies from the clinical development program for investigational gene therapy SRP-9001.
July 5, 2022
On July 5, 2022, Dyne Therapeutics announced that the U.S. Food and Drug Administration lifted the hold and cleared their Investigational New Drug application for DYNE-251.
June 30, 2022
On June 29, 2022, Santhera announced that the clinical module of its rolling new drug application (NDA) for vamorolone in Duchenne muscular dystrophy has been submitted to the U.S. Food and Drug Administration (FDA).
June 27, 2022
On Saturday, June 25, 2022, Italfarmaco Group shared a press release regarding positive topline data from its completed Phase 3 EPIDYS trial with Givinostat.
June 23, 2022
On June 23, 2022, Sarepta shared news regarding the MOMENTUM study.
Pliant Therapeutics
June 23, 2022
On June 22, 2022, Pliant Therapeutics announced the presentation of pre-clinical data from one of its developing drug candidates, PLN-101325.
Edgewise Duchenne News
June 21, 2022
On June 20, 2022, Edgewise Therapeutics announced positive results from the ARCH open label study of EDG-5506.