Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone fractures.
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 […]
Seasoned Healthcare Professional Brings Experience in Patient Advocacy and Community Education to Global Rare Disease Nonprofit Newport Beach, Calif. – January 22, 2024 – CureDuchenne, a global nonprofit committed to finding […]
Upcoming Events Across the Country Provide Latest Treatment Information and Resources for Managing Challenges of Living with Duchenne or Becker and Improving Quality of Life NEWPORT BEACH, Calif., January 22, […]
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