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BioMarin's combo therapy maintains walking and breathing in late-onset Pompe disease over 5 years

Company Fundamentals
02 Oct 2026
CNBC
View Source
Bullish
BioMarin's combo therapy maintains walking and breathing in late-onset Pompe disease over 5 years

BioMarin announced five-year Phase 3 trial results showing its combination therapy, Pombiliti and Opfolda, helped adults with late-onset Pompe disease maintain walking distance and stable respiratory function over continuous treatment. The study included 82 patients and found no new safety concerns. This data supports BioMarin's expanded role in rare metabolic diseases following its acquisition of Amicus Therapeutics, which marketed the therapy. The findings were presented at the World Muscle Society Congress, highlighting long-term benefits for this progressive muscle-weakening condition.

BioMarin's five-year Pompe disease data is the focus here, with Pluang's Health sector market snapshot as of Oct 02, 2026 23:13 WIB showing that out of 100 priced US Health stocks, 43 advanced while 55 declined. Notable movers include Oscar Health Inc (OSCR) at USD 30.70 with a 1-day change of +2.68% and Tenet Healthcare Corporation (THC) at USD 260.25 with +2.47%, while Sanofi SA (SNY) fell by -2.32% to USD 39.73, with typical hold times ranging from 15 to 94 days on Pluang.

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