Amicus Therapeutics’ Global Study on Pompe Disease: A Potential Game-Changer

Amicus Therapeutics ((FOLD)) announced an update on their ongoing clinical study.

Amicus Therapeutics is conducting a global observational study titled ‘A Global Prospective Observational Registry of Patients With Pompe Disease.’ The study aims to evaluate the long-term safety and effectiveness of treatments for Pompe disease, including their impact on quality of life and patient-reported outcomes. It also seeks to document the natural history of untreated Pompe disease.

The study involves various interventions, including enzyme replacement therapies (ERT) such as Cipaglucosidase alfa and Miglustat, as well as other ERTs like Alglucosidase alfa or Avalglucosidase alfa. It also includes untreated patients for comparison.

This 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.