We are studying a new gene therapy to see if it is safe and effective for males with Duchenne Muscular Dystrophy. The trial will assess improvements in muscle function and other health measures after a single intravenous dose.
Health conditions and diseases that the clinical trial is designed to study and treat.
Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle weakness and degeneration, primarily affecting boys. It is caused by mutations in the dystrophin gene, leading to difficulties in movement and muscle function.
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.