PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics Provides Key Regulatory Updates
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients
Edgewise Therapeutics announced the launch of an educational website dedicated to Becker muscular dystrophy
As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.
Capricor Therapeutics Announces Continuation of Phase 3 HOPE-3 Trial of CAP-1002 in Duchenne Muscular Dystrophy Based on Completion of Interim Futility Analysis
Agamree (vamorolone) was recently approved by the FDA, and Catalyst explains the new steroid and how to get it in this CureDuchenne Webinar.