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Rapport annuel- registre belge des maladies neuromusculaires 2015
particularity of a registry dedicated to neuromuscular disorders (compared to other health care registries) is that the patients’ population is heterogeneous. Neuromuscular disorders comprise indeed different ...
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 ...