Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting candidate declaration for GB703, a novel, ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

REGENXBIO touted data from the Phase III portion of a study that showed higher benefit that Elevidys.

(Reuters) -Capricor Therapeutics said on Monday the U.S. Food and Drug Administration (FDA) plans to convene a panel of outside experts before deciding on the company's cell therapy for a heart ...

Delandistrogene moxeparvovec-rokl shows significant motor function improvements in 8- and 9-year-olds with DMD, countering expected motor decline. Statistically significant differences were observed ...

Capricor Therapeutics has fallen out with its commercial partner for Duchenne muscular dystrophy (DMD) cell therapy ...

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 ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Investigational deramiocel is back under FDA review again ...

- PBGENE-DMD is a first-in-class in vivo gene editing approach for the majority of Duchenne Muscular Dystrophy patients impacted by dystrophin mutations in the most common ‘hot spot’ region between ...

Duchenne muscular dystrophy (DMD) causes muscle weakness that becomes more severe over time. While there is no cure, treatments can help slow the progression and manage symptoms and complications.

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...