A new rare muscle disorder has been identified by researchers at Ruhr-Universität Bochum (RUB). This hereditary disease is caused by a defect in the BICD2 gene that manifests itself in altered ...

Congenital muscular dystrophies (CMDs) comprise a heterogeneous group of inherited disorders primarily affecting muscle integrity and function, often with accompanying brain and ocular abnormalities.

Shares of Edgewise Therapeutics EWTX were up more than 18% on Monday after it announced positive results from the mid-stage CANYON study on its lead pipeline drug, sevasemten, in patients with a rare ...

A recent study has found that the SMCHD1 protein plays a key role in controlling how genes are processed, which affects the progression of Facioscapulohumeral Muscular Dystrophy (FSHD). This discovery ...

The U.S. Food and Drug Administration has approved the first generic version of Emflaza (deflazacort) oral suspension for Duchenne muscular dystrophy (DMD). Approval of the generic version of Emflaza ...

On Tuesday, Wave Life Sciences Ltd. (NASDAQ:WVE) revealed interim data from the ongoing Phase 2 FORWARD-53 study of WVE-N531 for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Regenxbio filed a lawsuit in 2020 alleging that Elevidys infringed a gene-therapy patent that the company licenses from University of Pennsylvania. ・The U.S. Court of Appeals for the Federal Circuit ...

A study of adults aged 60 years and older found that the use of statin therapy as primary cardiovascular disease (CVD) prevention was effective for preventing CVD and all-cause mortality, even in ...