TREAT-NMD patient registries - information for clinicians
TREAT-NMD is currently setting up registries for patients with Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). Other diseases may also have registries in future. Certain rare neuromuscular diseases will have their own rare disease registry - click here for details.
The primary objective of these registries is set up a single, global source of information that will facilitate international clinical trials for these diseases. When a patient registers with a national database, his/her medical data (including details of the precise genetic mutation causing the disease) is stored in a secure fashion in the country in question. Encrypted data is then transferred to the TREAT-NMD global registry where access will be available to international researchers upon request.
Access rules for researchers and industry partners are regulated in the TREAT-NMD registries charter, and further details are provided here. If researchers believe a patient meets the criteria for a trial and might benefit from participation, they will contact the person responsible for the registry in the respective country. The principal investigator can then de-code the data and forward information on the trial to the patient. Neither the patient's name nor any other personal information will be given to these researchers.
Patients who register with a TREAT-NMD registry will receive regular information on the TREAT-NMD projects and on research relating to DMD and SMA, plus up-to-date advice from international experts on standards of care and the chance to participate in multi-centre clinical trials.
If you have a DMD or SMA patient that you would like to register in the TREAT-NMD global registry, you should contact your national registry - click here for details. There are basically two methods used by participating national registries to collect data. Some registries (e.g. the UK and Germany) use self-report forms which can be filled out by the patients themselves, while others (e.g. France) use the so-called UMD forms which are filled out by clinicians or geneticists. Clicking on a country's flag will take you to more detailed information about the system used in that country. Feel free to contact the named national contact for further information. Alternatively, you can contact Professor Hanns Lochmüller, leader of the TREAT-NMD registries initiative.
