✉news WorldUS Politics first seen 6 h ago, last 3 h ago, peak #20
BioMarin Presents Five-Year Phase 3 Data for Pombiliti Therapy
Original: 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.
Why now: New long-term clinical data on a rare disease therapy was just presented at a major neuromuscular congress, drawing attention from the medical and patient communities.
BioMarinPombilitiOpfoldaWorld Muscle Society
Evidence
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