Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).

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

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Your DMD gene provides the instructions for making dystrophin, a protein essential to the strength and function of muscle fibers. In DMD, lack of dystrophin leads to progressive muscle ...

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

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

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

As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its ...

Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin ... to produce the protein known as dystrophin, it is this protein that ...