TREAT-NMD patient registries
A unified global source of information about neuromuscular patients
Scientific advances over recent years have lead to substantial changes in the treatment of many neuromuscular diseases. 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 diseases target specific genetic 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 of ensuring this can happen is to make sure that patients' details are all collected together 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 their disease.
The TREAT-NMD network is creating this kind of registry in countries across Europe and is also linking with other national registry efforts worldwide. To begin with we are focusing on registries for DMD and SMA, but we welcome contact from people interested in setting up other registries. As well as each national registry, we are also creating a single global registry which will combine the information from each of the national registries, and this will ensure that patients who register in their national registry can be contacted if their profile fits a clinical trial. In addition, these registries will help researchers to answer questions such as how common the individual diseases are across the world and will support other activities to improve patient care, such as the assessment of care standards in different countries.
Pharmaceutical companies interested in locating patients for a clinical trial will be able to request information from the TREAT-NMD global registry in a secure fashion that means that patients' details are protected but companies with legitimate interests can speedily find the information they need. All of this will help ensure that promising new treatments can be brought to patients as quickly as possible.
The registries pages here contain a lot of information for patients, for pharmaceutical companies, for researchers and for people interested in setting up a registry where none yet exists. Please use the links below to select the area you are most interested in.
Registries for DMD and SMA
Other information
The registry for rare neuromuscular disorders >>
In addition to DMD and SMA registries, patient registries are currently being developed for additional rare neuromuscular disorders. Find out about where these registries are, what they do and how to get involved.
How to set up a registry - the registry toolkit >>
If you are a patient organisation or specialist thinking of setting up a registry in a country where there is no TREAT-NMD registry or for a disease that TREAT-NMD doesn't yet address, this section is for you. Most of these efforts face many of the same challenges that we faced - such as legal and ethical framework, data protection, patient information and consent, professional involvement etc. By providing information about how we addressed some of these questions ourselves, we hope to facilitate and harmonize these efforts.
