Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Satellos Bioscience Inc. ("Satellos" or the "Company"), a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today ...

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

Capricor Therapeutics (CAPR) released expectation-beating results for the fourth quarter, sending the shares up over 5% in aftermarket trading.

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

The warning comes amid reports that unregulated therapies are being offered directly to patients, potentially illegally.View ...