It's a development that marks a potential breakthrough for people living with the rare and debilitating disease Duchenne muscular dystrophy (DMD)
KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB
Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne
Capricor’s BLA for Deramiocel received Priority Review in March 2025
The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment
Elevidys came under scrutiny in June when the FDA disclosed two fatal cases in non-ambulatory boys who developed dangerously high liver enzyme levels
The acquisition will follow the planned separation of Avidity’s early-stage precision cardiology programs into a new, independent company
Deflazacort oral suspension is indicated for treating Duchenne Muscular Dystrophy in patients 5 years of age and older
FDA requests Sarepta Therapeutics to suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths
Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older
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