World Muscle Society Annual Congress (WMS)

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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.

Thomas Crawford, MD (Credit: Johns Hopkins Medicine)

Higher Dose of Nusinersen Shows Greater Impact on Spinal Muscular Atrophy in Phase 2/3 DEVOTE Study

Isabella Ciccone, MPH
December 1st 2024

Findings from a phase 2/3 study demonstrated the efficacy and safety of a higher dose regimen of nusinersen in treating spinal muscular atrophy in both treatment-naïve and previously treated patients.

Duchenne Gene Therapy Shows No Alteration of Cardiac Outcomes in Phase 3 EMBARK Study

Duchenne Gene Therapy Shows No Alteration of Cardiac Outcomes in Phase 3 EMBARK Study

Marco Meglio
October 10th 2024

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.

Early Insights on 3 Patients With DMD From Trial Assessing Gene Therapy RGX-202: Aravindhan Veerapandiyan, MD

Early Insights on 3 Patients With DMD From Trial Assessing Gene Therapy RGX-202: Aravindhan Veerapandiyan, MD

Aravindhan Veerapandiyan, MD
October 17th 2023

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

Antisense Oligonucleotide AOC 1001 Demonstrates Long-Term Safety Open-Label Extension of MARINA Trial

Marco Meglio
October 15th 2023

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

Phase 1/2 Findings Highlight RGX-202’s Safety, Impact on Key Duchenne Biomarkers

Isabella Ciccone, MPH
October 6th 2023

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.

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