The team in Newcastle are experienced in the set-up and coordination of neuromuscular registries. A number of national and international registries are run from the John Walton Muscular Dystrophy Research Centre, collecting data on over 1,500 neuromuscular patients.
Patient registries refer to databases that collect information on people affected by a specific disease. When planning a clinical trial or research study it is important to reduce any possible barriers to therapy development. Finding enough patients that might be eligible to participate in trials for rare diseases can take years without a patient registry, delaying the testing of potential therapies. Patient registries provide an effective solution to this problem, ensuring that eligible patients can be found and contacted quickly. Huge advancements have been made in the treatment of many neuromuscular diseases with several new therapeutic strategies being developed. The primary aim of the registries that are coordinated from the John Walton Centre in Newcastle is to facilitate and accelerate research into rare inherited neuromuscular conditions. Patients’ clinical and genetic information are collected and made easily available for the researchers. Patients are at the centre of the research process and it is important that registered patients are kept informed of any research updates through regular newsletters. In addition to the research implications, registries can also be used to help provide important information to improve care standards.