IPS HEART Aims to Restore Full-Length Dystrophin with Two Regenerative Cell Therapies for Duchenne Muscular Dystrophy
IPS HEART has announced another important regulatory milestone after receiving FDA Rare Pediatric Disease Designation for ISX9-CPC, its investigational cardiac muscle therapy for Duchenne...
GEN6050X Exon 50 Skipping Base Editing Drug: One-Year Results Show Encouraging Progress for Duchenne Muscular Dystrophy
The GEN6050X exon 50 skipping base editing drug, developed by GenAssist Therapeutics, is attracting growing attention as one of the first base editing therapies...
Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen, an Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy (DMD)
Families affected by Duchenne muscular dystrophy (DMD) received encouraging news as Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted...
Santhera Expands Biomedica Agreement to Extend AGAMREE (Vamorolone) Distribution Across Central Asia and the Caucasus
Santhera today announces an expansion of its exclusive distribution agreement with Biomedica for AGAMREE (vamorolone), extending the partnership to include Georgia, Kazakhstan, Belarus, Azerbaijan...
SAT-3247 Six-Month TRAILHEAD Results Show Reduced Muscle Fat and Lower CK in Adults with Duchenne muscular dystrophy
New six-month interim results from the TRAILHEAD clinical trial suggest that SAT-3247 may help improve muscle health and preserve physical function in adults living...