Background and Purpose

What is the patient registry?

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, studies involving patients from more than one country are already in place.

Clinical trials for Myotonic Dystrophy can only include patients that meet specific inclusion criteria. When a clinical trial opens to recruitment, it is important that patients suitable for that trial can be invited to take part.. The best way to ensure this is to make sure that patients’ details are collected in a single database or “registry” that contains all the information needed to match patients with inclusion criteria. Capturing genetic report details and other key information about how the disease affects people on both an individual and group level over time is also very important for scientific research and assessing standards of care.

All patients who register and update their information will be contacted if their profile fits the requirements for a clinical trial, and they have not opted out. In addition, the registry will help researchers answer questions such as how common disorders like Myotonic Dystrophy are internationally, and will support other activities to improve patient care, and establish a standard of care worldwide.