Insmed’s gene therapy poised to challenge DMD landscape after IND clearance
Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...
PMLiVE3h
Santhera’s Duchenne muscular dystrophy drug Agamree accepted by SMC
Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...
Muscular Dystrophy News2h
DYNE-101 is leading to functional gains for DM1 patients in trial
DYNE-101 eased myotonia and led to other functional improvements for people with myotonic dystrophy type 1 (DM1) in a ...