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Amicus commercializes Galafold (migalastat) for Fabry disease, and Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for late-onset Pompe disease; positive long-term Phase 1/2 and pivotal Phase 3 data for AT-GAA program in Pompe disease[3]. Strong pipeline in gene therapies for rare diseases[2].
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