Background: Duchenne muscular dystrophy (DMD) is the most commonly inherited neuromuscular disease.\nTherapeutic agents for the treatment of rare disease, namely ââ?¬Å?orphan drugsââ?¬Â, have recently drawn the attention of\nresearchers and pharmaceutical companies. To ensure the successful conduction of clinical trials to evaluate novel\ntreatments for patients with rare diseases, an appropriate infrastructure is needed. One of the effective solutions for\nthe lack of infrastructure is to establish a network of rare diseases.\nMethods: To accomplish the conduction of clinical trials in Japan, the Muscular dystrophy clinical trial network\n(MDCTN) was established by the clinical research group for muscular dystrophy, including the National Center of\nNeurology and Psychiatry, as well as national and university hospitals, all which have a long-standing history of\nresearch cooperation.\nResults: Thirty-one medical institutions (17 national hospital organizations, 10 university hospitals, 1 national center,\n2 public hospitals, and 1 private hospital) belong to this network and collaborate to facilitate clinical trials. The Care\nand Treatment Site Registry (CTSR) calculates and reports the proportion of patients with neuromuscular diseases in\nthe cooperating sites. In total, there are 5,589 patients with neuromuscular diseases in Japan and the proportion of\npatients with each disease is as follows: DMD, 29 %; myotonic dystrophy type 1, 23 %; limb girdle muscular\ndystrophy, 11 %; Becker muscular dystrophy, 10 %. We work jointly to share updated health care information and\nstandardized evaluations of clinical outcomes as well. The collaboration with the patient registry (CTSR), allows the\nMDCTN to recruit DMD participants with specific mutations and conditions, in a remarkably short period of time.\nConclusion: Counting with a network that operates at a national level is important to address the corresponding\nnational issues. Thus, our network will be able to contribute with international research activity, which can lead to\nan improvement of neuromuscular disease treatment in Japan.
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