2023 Volume 40 Issue 1 Pages 8-11
Remudy (REgistry of MUscular DYstrophy) is a patient registry started in 2009 in collaboration with TREAT–NMD, an international clinical research network, to promote clinical development of hereditary neuromuscular diseases. In close collaboration with the Japan Muscular Dystrophy Association, a patient organization, the registry office was established at the National Center of Neurology and Psychiatry, and the registry started with dystrophinopathies, and is now operating registries for GNE myopathy, myotonic dystrophy, Facioscapulohumeral muscular dystrophy, and congenital muscular disorders. It has conducted various epidemiological studies, contributed to clinical trial feasibility studies and patient recruitment, and disseminated information to patients. In recent years, the use of real–world data in therapeutic development has been attracting attention, and among these, the utilization of the registry under the pharmaceutical system is being promoted under the Clinical Innovation Network. It is hoped that the registry will be widely utilized in areas ranging from therapeutic development to safety monitoring activities.
