Shares of Sarepta Therapeutics SRPT plunged more than 20% yesterday after the company reported the death of a patient after ...
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 ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...
More than 800 people have been treated with Elevidys, the only approved treatment for Duchenne muscular dystrophy, the company said.