We are testing a new gene therapy for boys with Duchenne Muscular Dystrophy who can walk. The study aims to find the right dose and evaluate the therapy's safety and effectiveness compared to a placebo.
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 mobility.
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..
Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.
Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.
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.