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- W2313395964 abstract "The development of orphan medicines presents many challenges. Clinical trials with new therapeutic strategies are now being planned and conducted for many orphan diseases such as Duchenne and Becker muscular dystrophy (DMD/BMD). However, since adequate numbers of patients are needed to achieve significant results for clinical trials, patient registries are an important infrastructure worldwide, especially in the case of rare diseases. We developed a national registry of Japanese DMD/BMD patients in collaboration with European research network, TREAT-NMD. Japanese registry adopts a self-report system and this database includes clinical data and molecular genetic data. The main purpose of this registry is the effective recruitment of eligible patients for a clinical trial and may provide timely information to individual patients. The registry data gives us more detailed knowledge of natural history, epidemiology, and clinical care. This registry will gain trial readiness in Japan and accelerate more effectively harmonization with other countries to fight orphan diseases." @default.
- W2313395964 created "2016-06-24" @default.
- W2313395964 creator A5084693390 @default.
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- W2313395964 date "2011-01-01" @default.
- W2313395964 modified "2023-09-30" @default.
- W2313395964 title "Registry of muscular dystrophy (Remudy). Construction of the patient self-report registry and collaboration with overseas network" @default.
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- W2313395964 doi "https://doi.org/10.5692/clinicalneurol.51.901" @default.
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- W2313395964 hasPublicationYear "2011" @default.
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