What are the TREAT-NMD Global Registries?
New therapeutic strategies for neuromuscular diseases are being developed and some of these treatments will require large studies involving patients from more than one country. TREAT‑NMD has created a Global Registry for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) with trials and development of innovative therapies in mind. Global registries for other conditions are in development.
National DMD and SMA registries feed into a single global registry which combines the information and ensures that patients who register in their national registry can be contacted if their profile fits the criteria for a clinical trial. All national registries that are part of the TREAT-NMD Global Registries collect as a minimum a standardized core set of information that are not only useful for planning clinical trials but also recruiting patients into them.
These registries help researchers to answer questions such as how common the individual diseases are across the world and support other activities to improve patient care, such as the assessment of care standards in different countries.
The TREAT-NMD Global Registries are governed by a Charter and the TREAT-NMD Global Database Oversight Committee (TGDOC). The TGDOC is responsible for reviewing the requests for data from the Global Registry and it votes on whether the request is in line with the Global Registries charter and in patients’ best interest.
Researchers and pharmaceutical companies interested in locating patients for a clinical trial are able to request information from the TREAT-NMD Global Registry meaning that they can speedily find the information they need.
Patients who may be eligible for a clinical trial will be informed through their own national registry and patients’ details are never disclosed directly to the researcher/company. If a patient wishes to take part in a clinical trial then they will be asked to contact their local trial site, information for which will be provided on any correspondence from the national registry curator, and not the company.
All of this will help ensure that promising new treatments can be brought to patients as quickly as possible.