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 ...
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 ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...
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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