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Rapport annuel- registre belge des maladies neuromusculaires 2015
rare diseases affecting the peripheral nerves, or the muscles, or the neuromuscular junction, or anterior horn cells. Some of those diseases are genetic, while others are not. The vast majority of them ...
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
Databases Development Diagnostics disease Diseases Duchenne muscular dystrophy electronic Frequency Genetic Genetic Therapy global im Institute IS journal Medicine Muscular Dystrophies Muscular dystrophy ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
POPULATION prevalence protein Rare disease REGISTRIES Registry successful Therapy UK utilization Abstract: Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the ...