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Population
Patients with Duchenne muscular dystrophy from the STRIDE Registry and CINRG DNHS
Comparison
Ataluren plus standard of care vs standard of care alone
Design
Registry and natural history study comparison
Observational data associate ataluren with delayed DMD progression; leaves open need for randomized confirmation before practice change.
Investigators from Europe and the USA, representing the STRIDE Registry and Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), examined the effectiveness of ataluren and standard of care in the Registry versus stand of care alone in the CINRG DNHS.
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Sameh S Morkous (2020) studied this question.
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