We are testing a new intravenous treatment for adults with late-onset Pompe disease to evaluate its safety and potential benefits. This study will help us understand how well the treatment works and how it is tolerated.
Health conditions and diseases that the clinical trial is designed to study and treat.
Pompe Disease is a rare genetic disorder caused by the buildup of glycogen in the body's cells due to a deficiency of the enzyme acid alpha-glucosidase. This can lead to muscle weakness, respiratory issues, and other serious health 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.