Amicus Therapeutics Unveils Promising 4-Year Data for Pombiliti® + Opfolda® in Late-Onset Pompe Disease Study at ICIEM
Reuters
Sep 08
Amicus <a href="https://laohu8.com/S/LENZ">Therapeutics</a> Unveils Promising 4-Year Data for Pombiliti® + Opfolda® in Late-Onset Pompe Disease Study at ICIEM
Amicus Therapeutics Inc. has announced new 4-year data from the PROPEL open-label extension study on the efficacy of Pombiliti® (cipaglucosidase alfa-atga) and Opfolda® (miglustat) in adults with late-onset Pompe disease (LOPD). The results, which were presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, highlight sustained improvements in muscle function, muscle strength, and biomarker endpoints in patients. Additionally, various abstracts related to Pombiliti and Opfolda, including studies comparing their efficacy and safety to alglucosidase alfa, as well as real-world impacts of the treatment switch, were presented at the congress. These findings contribute to the growing body of evidence supporting the clinical profile of the treatment combination for LOPD.
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