Based on the positive findings from the Phase 1/2 study, Genethon is planning to launch Phase 3 testing of GNT0004 next year.

Columnist Robin Stemple knows his walker should help prevent falls, but it didn't work out that way on a simple maneuver to ...

Columnist Patrick Moeschen, who lives with limb-girdle muscular dystrophy, explains why controlling what he can is so ...

Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...

Regenxbio hopes to use the study's results to ask the U.S. Food and Drug Administration to grant accelerated approval to the ...

As a caregiver for three sons with DMD, columnist Betty Vertin clung to her many responsibilities. This year she's learned to let go a bit.

The U.S. Food and Drug Administration (FDA) has given Atamyo Therapeutics the go-ahead to start a Phase 1b clinical trial to test ATA-200 — the company’s gene therapy candidate for limb-girdle ...

A new technology called StitchR effectively restored the production of large muscle proteins dystrophin and dysferlin in mouse models of Duchenne muscular dystrophy (DMD) and limb-girdle muscular ...