World Muscle Society Annual Congress (WMS)

After 52 weeks of treatment, no significant differences were found in cardiac MRI measures such as ejection fraction, end diastolic volume, or circumferential strain between the two groups.

The pediatric neuromuscular neurologist at Arkansas Children's Hospital and associate professor of pediatrics at the University of Arkansas for Medical Sciences talked about findings from a phase 1/2 study on dosing 3 pediatric patients with Duchenne muscular dystrophy with REGENXBIO’s RGX-202. [WATCH TIME: 4 minutes]

AOC 1001 consistently demonstrated directional improvements across multiple aspects of myotonic dystrophy type 1, including measures of myotonia, strength, function, and patient reported outcome.

RGX-202, a gene therapy for Duchenne muscular dystrophy, was well-tolerated with no therapy-related serious adverse effects in 3 patients who received the level 1 dosage.