Biopharmaceutical Company
Appears in 2 stories · Last updated Oct 5, 2026
As of October 2026: Markets Pombiliti and Opfolda for late-onset Pompe disease
Adults with late-onset Pompe disease who had already spent an average of 7.6 years on enzyme therapy kept their walking distance nearly unchanged over five more years on BioMarin's Pombiliti and Opfolda, while lung function slipped slightly. BioMarin presented the results October 2 at the World Muscle Society congress in Hiroshima.
Updated Oct 4
As of May 2026: Market leader with only approved achondroplasia treatment
About 250,000 people worldwide have achondroplasia, the most common genetic cause of dwarfism. Until BioMarin's Voxzogo, the only approved treatment required daily injections.
Updated May 29
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