A Pfizer gene therapy for the rare muscle-wasting disease Duchenne muscular dystrophy did not help patients’ ability to walk or stand up in a pivotal clinical trial. The pharmaceutical giant is still ...
Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting a conditional marketing authorization for Duvyzat (givinostat, Italfarmaco) to treat Duchenne muscular dystrophy ...
A patient has died after receiving Sarepta Therapeutics’ approved gene therapy for Duchenne muscular dystrophy — the second fatality reported by the company in the past three months. Like the first ...
With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. | Late Thursday, PTC revealed that ...
The FDA accepted the resubmitted NDA for ataluren, a treatment for nmDMD, without an action date due to a previous complete response letter. Study 041 and the STRIDE registry data support ataluren's ...
Duchenne muscular dystrophy (DMD) is a genetic condition that causes increasing muscle weakness over time. While DMD currently has no cure, researchers are studying potential new treatments. When ...
PARAMUS, N.J., March 10, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that ...
A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at ...
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