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BioMarin Pharmaceutical Presents New Efficacy Data On POMBILITI + OPFOLDA In Pompe Disease At 31st Annual Congress Of World Muscle Society; No New Safety Signals Identified

Oct 2, 2026 · 11:01 AM ET· updated 1h ago

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented

Why It Matters

BioMarin showed new proof that their Pompe disease medicine works well. No new safety worries came up, which is good news.

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