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

The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...

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

It’s caused by mutations in the DMD gene, which carries the instructions for making the protein dystrophin. Approximately 70% to 80% of people with DMD inherit a genetic mutation that causes the ...

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

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

Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy.

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene ... for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making ...

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

But when he was 7 months old, Colton Belluzzo was diagnosed with Duchenne muscular dystrophy — a rare ... essentially placed a copy of the dystrophin gene into a virus, and then injected that ...