Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)
Meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT.
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy Treatment.
PTC Therapeutics Provides Key Regulatory Updates
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
Dyne Therapeutics will present initial clinical data, first released on January 3rd, 2024, from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
The FDA will make a decision whether or not to grant full approval of Sarepta’s gene therapy, Elevidys, by June 21,2024.
REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and […]
