Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...

Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

Myotonic dystrophy is an inherited muscular dystrophy causing muscle weakness and wasting. Excessive daytime sleepiness (hypersomnia) is a frequent complaint of people with myotonic dystrophy and is ...

a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

DELIVER Trial of DYNE-251 in DMD In September 2024, Dyne reported positive efficacy data and a favorable safety profile 1 from the ongoing Phase 1/2 global DELIVER trial of DYNE-251 in males with ...

CHARLESTOWN, Mass., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company” or "Solid”), a ...

Presented additional data from the Phase 1 clinical trial ENTR-601-44-101 and new preclinical data supporting ENTR-601-45 for DMD at 2024 World ...

“It is not the fact finder. It may apply facts to the law, but it does not rule on them.” In late May, the court released an opinion that refers to a separate case involving Kennedy and ...