We are evaluating the safety and effectiveness of a new treatment for children with late-onset Pompe disease. This study aims to understand how well the medications work together and their effects on young patients.
Health conditions and diseases that the clinical trial is designed to study and treat.
Late-onset Pompe disease is a rare genetic disorder that affects the muscles and heart, caused by the buildup of glycogen due to a deficiency of the enzyme acid alpha-glucosidase. Symptoms may include muscle weakness, respiratory issues, and heart problems.
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.