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.

A groundbreaking study, recently published in Nature Communications, sheds light on a newly identified subtype of muscular dystrophy, revealing an unsuspected role of SNUPN gene in muscle cell ...

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 ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

Celine Dion announced Friday she has canceled her upcoming world tour as the five-time Grammy Award winning singer tries to “build back strength” following her diagnosis with stiff-person syndrome, a ...

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 ...

Researchers at University of North Carolina at Chapel Hill have gotten a glimpse into what happens in patients suffering from Duchenne Muscular Dystrophy, the most common and severe form of the ...

Oct 31 (Reuters) - Sarepta Therapeutics shares fell nearly 44% on Tuesday, after its gene therapy for a progressive muscle-wasting disorder failed to meet the main goal in a late-stage trial that was ...

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 ...