Information for doctors
The registry for patients with a Myotonic Dystrophy collects genetic and clinical data about patients with DM1 or DM2/PROMM. For research into treatments for DM1 or DM2, it is important that the researchers have precise information about the genetic mutation that causes the condition. This information will be collected by the registry and validated by experts. In anonymous form, the valuable medical data collected will be made available to selective researchers around the world, thereby accelerating the research into Myotonic Dystrophy type 1 and type 2.
Additionally, with the advent of clinical trials and research studies for some of these neuromuscular conditions, patients who might be eligible for certain clinical trials or research studies must be readily identifiable. This is what patient registries can offer: they contain accurate and up-to-date information about the patients’ genetic mutations and clinical conditions, as well as their contact details. Without a patient registry for rare conditions such as Myotonic Dystrophies, it could take years to find enough patients for a meaningful trial – and this could significantly delay the development of potential therapies.
In the DM patient registry this information is both provided by the patient and professionals involved in the patient’s care after full consent obtained by the patient.