We are testing a new gene therapy called UX111 for individuals with Mucopolysaccharidosis IIIA. The goal is to see if it is safe and effective in treating this condition.
Health conditions and diseases that the clinical trial is designed to study and treat.
Mucopolysaccharidosis III (MPS III) is a rare genetic disorder that affects the metabolism of sugars, leading to a buildup of harmful substances in the body. This condition can cause various symptoms, including developmental delays, behavioral issues, and physical 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.