There are multiple clinical trials that are currently underway for Pompe disease. Because there are so many different trials, which is unique for a rare disease like Pompe, it is important to understand the clinical trial process, the trials phases, and other basic information.
Please find in the attachment a short overview of clinical trials in general.
The International Pompe Association
(courtesy of the AMDA – Acid Maltase Deficiency Association)
- Overview of Clinical Trials (PDF, 320.97 kB)