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FDA Approves Duvyzat (givinostat) for Duchenne Treatment
FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy Treatment.
PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics Provides Key Regulatory Updates
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver for Patients
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.
Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication
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
REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
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 […]
PTC Therapeutics – EMFLAZA® LANDSCAPE IN 2024
Join PTC Therapeutics and CureDuchenne for this prerecorded webinar to hear important information regarding Emflaza, PTC Cares and the programs that support the Duchenne community. Topics include:Understanding Your PrescriptionHow to Ensure Your Son […]
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.