An introduction from the original network coordinators
TREAT-NMD is a network for the neuromuscular field that is creating the infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch in January 2007 the network's focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide.
Recent years have seen rapid developments in the neuromuscular field and a corresponding surge in interest from the pharmaceutical industry. Promising preclinical results raise the potential for new therapies in the near future. Yet translational research towards therapy development for neuromuscular diseases has faced a number of barriers. For patients, promising research results have still not been translated into the treatments they hope for, while lack of standardized care guidelines prevents many from receiving optimal care. For the biomedical industry, identifying the investigators and sites with the relevant expertise and accessing the appropriate patient cohorts for clinical trials has been a significant challenge. For clinicians and researchers, lack of support tools such as validated clinical outcome measures or standard operating procedures for research protocols has held back therapeutic development. TREAT-NMD addresses all these issues, uniting the stakeholders in the community and providing an infrastructure that is accelerating research and therapy development, increasing collaboration, improving patient care and helping to support ‘clinical trial readiness’ on an international scale. TREAT-NMD was established as a EUfunded ‘network of excellence’ with the remit of ‘reshaping the research environment’ in the neuromuscular field. The network has developed from its European roots to become a global organization bringing together leading specialists, patient groups and industry representatives to ensure preparedness for the trials and therapies of the future while promoting best practice today.
Professors Kate Bushby and Volker Straub
Newcastle University, UK