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

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

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

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

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

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 ...

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with ...

Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could ...