Clinical trials for new therapeutic strategies are now being planned for 
Duchenne and Becker muscular dystrophies (DMD/BMD); however, many challenges 
exist in the planning and conduction of a clinical trial for rare diseases. The 
epidemiological data, total number of patients, natural history, and clinical 
outcome measures are unclear. Adequate numbers of patients are needed to achieve 
significant results in clinical trials. As solutions to these problems, patient 
registries are an important infrastructure worldwide, especially in the case of 
rare diseases such as DMD/BMD. In Europe, TREAT-NMD, a clinical research network 
for neuromuscular disorders, developeda global database for dystrophinopathy 
patients. We developed a national registry of Japanese DMD/BMD patients in 
collaboration with TREAT-NMD. The database includes clinical and molecular 
genetic data as well as all required items for the TREAT-NMD global patient 
registry. As of July 2011, 750 patients were registered in the database. The 
purpose of this registry is the effective recruitment of eligible patients for 
clinical trials, and it may also provide timely information to individual 
patients about upcoming trials. This registry data also provides more detailed 
knowledge about natural history, epidemiology, and clinical care. In recent 
years, drug development has become dramatically globalized, and global clinical 
trials (GCTs) are being conducted in Japan. It is appropriate, particularly with 
regard to orphan diseases, to include Japanese patients in GCTs to increase 
evidence for evaluation, because such large-scale trials would be difficult to 
conduct solely within Japan. GCTs enable the synchronization of clinical drug 
development in Japan with that in Western countries, minimizing drug approval 
delays.