The World Muscle Society Annual Congress is a community-driven meeting to promote, disseminate, and share all aspects of neuromuscular physiology and diseases, from basic science to patient care.
Early Insights on 3 Patients With DMD From Trial Assessing Gene Therapy RGX-202: Aravindhan Veerapandiyan, MD
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]
Antisense Oligonucleotide AOC 1001 Demonstrates Long-Term Safety Open-Label Extension of MARINA Trial
AOC 1001 consistently demonstrated directional improvements across multiple aspects of myotonic dystrophy type 1, including measures of myotonia, strength, function, and patient reported outcome.
Phase 1/2 Findings Highlight RGX-202’s Safety, Impact on Key Duchenne Biomarkers
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.
2 Clarke Drive Cranbury, NJ 08512