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 […]
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
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Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
The silver screen has been featuring more and more stories about people with disabilities, whether it be developmental, physical, or mental. With more representation in popular culture, comes more understanding […]