Treatment with Ataluren for Duchene Muscular Dystrophy
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.
Published In/Presented At
Morkous S. S. (2020). Treatment with Ataluren for Duchene Muscular Dystrophy. Pediatric neurology briefs, 34, 12. https://doi.org/10.15844/pedneurbriefs-34-12
Medicine and Health Sciences | Pediatrics
Peer Reviewed for front end display
Department of Pediatrics, Department of Pediatrics Faculty