BioMarin Reports 5-Year Motor, Lung Data For Pompe Disease Therapy

BioMarin reported five-year Phase 3 data for POMBILITI + OPFOLDA in adults with late-onset Pompe disease at the World Muscle Society Congress in Hiroshima. The long-term clinical-data update is a modestly positive development for the company’s rare-disease portfolio, though the article provides no efficacy, safety, or financial figures.