Amicus Therapeutics Inc. has announced the presentation of new data from clinical and real-world studies of Galafold® (migalastat) for Fabry disease and POMBILITI® (cipaglucosidase alfa) + OPFOLDA® (miglustat) for late-onset Pompe disease at the 22nd Annual WORLDSymposium™ 2026. The new data include results from an open-label study evaluating the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat in pediatric patients with Fabry disease, as well as analyses of real-world effectiveness of migalastat versus enzyme replacement therapy in treatment-naïve patients with Fabry disease. Additional presentations cover structured methodologies for evaluating patient-reported outcomes and clinical characterization of Fabry disease using Swedish national registers. These results are being presented during the conference.

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