Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
REGENXBIO Inc. (Nasdaq: RGNX) today 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 ...
Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD.
The company behind the drug, Sarepta Therapeutics, said that the patient suffered acute liver injury, a known side effect, ...
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