---
title: "Amicus Therapeutics Unveils New Clinical Data for Galafold and Pombiliti + Opfolda in Fabry and Pompe Diseases"
type: "News"
locale: "en"
url: "https://longbridge.com/en/news/274659985.md"
datetime: "2026-02-03T13:01:15.000Z"
locales:
  - [zh-CN](https://longbridge.com/zh-CN/news/274659985.md)
  - [en](https://longbridge.com/en/news/274659985.md)
  - [zh-HK](https://longbridge.com/zh-HK/news/274659985.md)
---

> Supported Languages: [简体中文](https://longbridge.com/zh-CN/news/274659985.md) | [繁體中文](https://longbridge.com/zh-HK/news/274659985.md)


# Amicus Therapeutics Unveils New Clinical Data for Galafold and Pombiliti + Opfolda in Fabry and Pompe Diseases

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. 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: GNW9647268-en) on February 03, 2026, and is solely responsible for the information contained therein. © Copyright 2026 - Public Technologies (PUBT)

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