Amicus Therapeutics' Pompe Disease Study: A Potential Game-Changer for the Company's Stock Performance

Amicus Therapeutics is conducting a global observational study on Pompe disease to evaluate the long-term safety and effectiveness of treatments, including enzyme replacement therapies. The study aims to gather real-world evidence on the treatments' safety and effectiveness and will follow a cohort model over five years. This study has the potential to influence Amicus Therapeutics' stock performance by validating the effectiveness of its treatments and positioning the company competitively within the industry.

Amicus Therapeutics (FOLD) has announced an update on its ongoing global observational study titled 'A Global Prospective Observational Registry of Patients With Pompe Disease.' This study aims to evaluate the long-term safety and effectiveness of treatments for Pompe disease, including enzyme replacement therapies (ERT) such as Cipaglucosidase alfa and Miglustat, as well as other ERTs like Alglucosidase alfa or Avalglucosidase alfa. The study also includes untreated patients for comparison.

The observational study follows a cohort model and is prospective in nature, meaning it will follow patients over time to collect data. The primary purpose is to gather real-world evidence on the treatments' safety and effectiveness. The study began on February 16, 2024, with an estimated completion timeline of five years. The latest update was submitted on August 11, 2025, indicating ongoing progress.

This study could influence Amicus Therapeutics' stock performance by potentially validating the effectiveness of its treatments, thereby boosting investor confidence. It also positions the company competitively within the industry, as real-world data becomes increasingly valuable.

The study is currently ongoing, with further details available on the ClinicalTrials portal.

References:
[1] https://www.tipranks.com/news/company-announcements/amicus-therapeutics-global-study-on-pompe-disease-a-potential-game-changer
[2] https://www.placera.se/pressmeddelanden/clinical-progress-in-rare-disease-signals-long-term-revenue-potential-for-innovators-20250806