Biotechnology company (acquired)
Appears in 1 story
Acquired by BioMarin in December 2025
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.
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