Amicus Therapeutics Unveils Promising 4-Year Data for Pombiliti® + Opfolda® in Late-Onset Pompe Disease Study at ICIEM

Reuters
2025/09/08
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.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Amicus Therapeutics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9525004-en) on September 08, 2025, and is solely responsible for the information contained therein.

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