Background and purpose

What is the patient registry and why do we want to create one?

Scientific advances over recent years have led to substantial changes in the treatment of many disorders. New therapeutic strategies are being developed and, for some of these treatments, plans for large studies involving patients from more than one country are already in place.

Several new therapeutic strategies for neuromuscular disorders like Myotonic Dystrophy type 1 target specific gene defects. When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly. The best way to ensure that this happens, is to make sure that patients’ details are all collected in a single database or “registry” that contains all the information that researchers will need, including each patient’s particular genetic defect and other key information about that disorder.

The TREAT-NMD network is creating this kind of global registry, which means that all patients who register will be contacted if their profile fits the requirements for the clinical trial. In addition, the registry will help researchers answer questions such as how common disorders like Myotonic Dystrophy type 1 are internationally, and will support other activities to improve patient care, and establish a standard of care worldwide.