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...
Biophytis’ BIO101 Therapy for Treatment of Sarcopenia Could Bring Hope to Duchenne Muscular Dystrophy Patients
The Phase 2 clinical trial SARA-INT was published in the Journal of Cachexia, Sarcopenia and Muscle (JCSM), the primary reference journal for sarcopenia research,...
Eteplirsen (Exondys 51) Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy
Following promising results in slowing heart function decline in patients with DMD, Eteplirsen (Exondys 51) continues to show potential in addressing key aspects of...