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
Nxera will take full responsibility for regulatory approvals, clinical development where required, manufacturing and commercialization in the licensed territories
The partnership also sets the stage for broader collaboration on rare disease treatments
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
Subscribe To Our Newsletter & Stay Updated
