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[Remudy]
En Kimura1, Harumasa Nakamura, Ichizo Nishino
1Translational Medical Center, National Center of Neurology and Psychiatry.
Brain and Nerve = Shinkei Kenkyu No Shinpo
|November 20, 2014
Summary
Remudy, a Japanese registry for muscular dystrophies like Dystrophinopathy and GNE myopathy, builds infrastructure to speed up clinical research and trials for rare neuromuscular diseases.
Area of Science:
- Neurology
- Rare Diseases
- Clinical Research Infrastructure
Background:
- Dystrophinopathy and GNE myopathy are rare neuromuscular diseases with limited clinical research.
- Developing effective treatments requires robust clinical research infrastructure.
Purpose of the Study:
- To establish a clinical research infrastructure for Dystrophinopathy and GNE myopathy.
- To accelerate clinical development research for these rare diseases.
Main Methods:
- Operated the Remudy (Registry of Muscular Dystrophy) national registries in Japan.
- Collaborated with the TREAT-NMD alliance.
- Utilized a 'registry-based' research style.
Main Results:
- Provided data sets for feasibility studies.
- Facilitated clinical trial recruitment by disseminating relevant information.
- Presented natural history and epidemiology data for rare diseases.
Conclusions:
- Remudy serves as a prototype model for clinical research infrastructure.
- This infrastructure aims to overcome challenges in rare and incurable neuromuscular diseases.
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