Pombiliti

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.