September 30, 2026 / Research

Catch up on the 31st Congress of the World Muscle Society

Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. The meeting offers a dynamic platform for sharing new data, fostering collaborations, and advancing the science behind therapies for diseases like Duchenne and Becker muscular dystrophy. This year’s event in Hiroshima, Japan, features talks focusing on biology and therapeutic options for myotonic disorders, drug-induced and infectious myopathies, and trust, transparency, and therapeutic understanding.

Below are highlights from the 2026 meeting, updated daily with information from members of PPMD’s team attending the meeting in Hiroshima:

Industry updates:

Sarepta Therapeutics Presents Safety and Functional Data for ELEVIDYS in Older Ambulatory Patients with Duchenne

Sarepta Therapeutics presented new data on ELEVIDYS, including findings in ambulatory individuals with Duchenne who were 8–12 years old when treated. Over two years, study participants who received ELEVIDYS showed durable and clinically meaningful benefits across measures of motor function, including the North Star Ambulatory Assessment (NSAA) and timed function tests, compared to a well-matched external control. Sarepta noted that functional benefits were observed at both one and two years after treatment, similar to previous results in patients who were treated between 4 and 7 years in age. 

Additional data presented at WMS included findings from the ENDEAVOR and ENVOL studies in younger children ages 2 to 3 years, showing robust ELEVIDYS micro-dystrophin expression and localization in muscle cells at 12 weeks after treatment—higher on average, Sarepta notes, than those previously reported in older ambulatory and non-ambulatory individuals.

Read Sarepta’s press release and community letter to learn more.

Capricor Therapeutics Presents HOPE-3 Open-Label Extension Data on Upper Limb Function

Capricor Therapeutics presented new 24-month data from the ongoing HOPE-3 open-label extension (OLE) study of Deramiocel. In the 24-month crossover analysis, patients who started Deramiocel after one year on placebo showed significantly slowed upper limb decline compared to their decline during the placebo period. Additionally, both groups showed slower decline in upper limb function compared to natural history at two years.

According to Capricor, the FDA has received these data as part of the recent major amendment to the Deramiocel Biologics License Application (BLA), which will be reviewed ahead of the November 22, 2026 PDUFA target action date.

Read Capricor’s press release to learn more.

Capricor will join PPMD on Wednesday, October 7, 2026 at 1:00 PM ET for a community webinar to provide an overview of this data and answer questions from the community. Register here and submit questions in advance here.

Stay tuned for more updates from WMS 2026

The 2026 World Muscle Society Congress is still underway, and PPMD’s team will continue following presentations and sharing key updates throughout the meeting. Check back here for new information and developments from Hiroshima, including updates on Duchenne and Becker research, clinical care, and therapeutic advances.

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