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World Muscle Society
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- 1BioMarin Presents Five-Year Phase 3 Data for Pombiliti Therapy▼BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress
BioMarin announced five-year Phase 3 data for Pombiliti (cipaglucosidase alfa-atga) combined with Opfolda (miglustat), presented at the World Muscle Society Annual Congress. The long-term results concern the company's enzyme replacement therapy for late-onset Pompe disease, and the announcement was issued via press release. The new data points were shared with clinicians and researchers attending the congress.
- 2Sarepta Reports New ELEVIDYS Data at World Muscle Society Congress▼Sarepta Therapeutics Presents New Data at 2026 World Muscle Society Annual Congress Demonstrating Safety and Clinically Meaningful Functional Benefit of ELEVIDYS (delandistrogene moxeparvovec) in Older Ambulatory Patients with Duchenne
Sarepta Therapeutics presented new findings at the 2026 World Muscle Society Annual Congress showing that ELEVIDYS (delandistrogene moxeparvovec) is safe and delivers clinically meaningful functional benefit in older ambulatory patients with Duchenne muscular dystrophy. The data extend the gene therapy's evidence base beyond younger patient groups, a question that has long been debated by clinicians and regulators in the Duchenne community.
- 3Five-Year Data on Duchenne Drug Brogidirsen Presented at Muscle Congress●Brogidirsen (NS-089/NCNP-02) 5-Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2026 World Muscle Society Congress
Five-year clinical trial data for brogidirsen (NS-089/NCNP-02), an investigational treatment for Duchenne muscular dystrophy, has been presented at the 2026 World Muscle Society Congress. The long-term results are being shared with specialists in neuromuscular medicine, offering the fullest picture yet of the drug's safety and effectiveness over five years in patients with the muscle-wasting disease.