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 ...

The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular ...

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 FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...