TREAT-NMD, the international network for neuromuscular diseases, is a key tool for many people affected by these diseases, as it aims to optimise knowledge and accelerate treatment. Within TREAT_NMD there is the Duchenne muscular dystrophy (DMD) registry, which contains information on over 13,500 patients from 31 different countries. The information contained is in the form of standardized patient registries, established in individual countries, through which clinical outcomes and new technologies can be evaluated. An article published in Human Mutation describes how the national TREAT-NMD registries for DMD were established. The authors explore how the growth and accessibility of TREAT-NMD has fostered collaboration between academia, patient associations, and industry, as well as a marked increase in the feasibility of clinical trials, which in turn has led to the development of potential treatment strategies for DMD, including exon skipping therapy. In addition, the authors believe that registries offer tools for evaluating other therapies such as corticosteroids. According to the authors, "the establishment of harmonized national patient registries and an international registry of DMD patients has created a centralized point of access to information (which), in turn, has served to increase interest from TREAT-NMD's industry partners." The authors also note that "the establishment of harmonized national patient registries and an international registry of DMD patients has created a centralized point of access to information (which), in turn, has served to increase interest from TREAT-NMD's industry partners.
These registries have contributed to improved patient care (CARE-NMD is an example) and have been instrumental in research and health policies in many countries. The authors further argue that the impact on national registries will be further enhanced through their continued inclusion in the Global DMD Registry http://www.treat-nmd.eu/resources/patient-registries/overview/. The article also highlights the challenges that TREAT-NMD faces, such as the fact that to date not all national registries have transferred their data to the global registry. Finally, the authors also illustrate problems with grants, whereby some registries have had to rely on short-term funding while waiting to identify alternative sources of funding.