RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

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

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

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.

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

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

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 ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

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