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, prospective observational registry 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, assess their impact on quality of life, and describe the natural history of untreated Pompe disease. This research is significant as it provides real-world data on patient outcomes.

The study involves several interventions, including Cipaglucosidase alfa and Miglustat, both used as enzyme replacement therapies (ERTs) for treating Pompe disease. These interventions aim to improve patient outcomes by replacing deficient enzymes in affected individuals.

The study is observational, following a cohort model with a prospective time perspective. It does not involve random allocation or masking, as its primary purpose is to gather real-world data over a five-year period.

The study began on February 16, 2024, with an estimated completion date yet to be determined. The most recent update was submitted on August 11, 2025. These dates are crucial for tracking the study’s progress and ensuring timely updates for stakeholders.

The market implications of this study are significant for Amicus Therapeutics, as positive findings could enhance investor confidence and potentially boost stock performance. The study’s outcomes may also influence the competitive landscape of Pompe disease treatments, impacting other companies in the industry.

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