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 initially established as a EU funded ‘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.