Regenxbio Reports Positive Biomarker Data From Affinity Duchenne Trial Of Rgx-202 Gene Therapy
REGENXBIO reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated...
Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Satellos Bioscience, a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced initial Phase...
Precision BioSciences Presents Preclinical Efficacy and Durability Data on PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) at the 2025 Muscular Dystrophy Association...
This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up...
Sarepta, Reports 16 Years Old Boy Death After Elevidys Gene Therapy Treatment
On Tuesday, Sarepta Therapeutics said that the first recorded fatality associated with their gene therapy for Duchenne muscular dystrophy, Elevidys, had occurred in a...
Givinostat’s Exclusive Distribution Agreement in 17 Central and Eastern European Countries, Including the Baltic States, has been Announced by Italfarmaco and Medis
Italfarmaco and Medis, a leading pharmaceutical commercialisation company in Central and Eastern Europe, today announced an exclusive distribution agreement for Givinostat (Duvyzat), Italfarmaco’s drug...