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Ataluren in nonsense mutation Duchenne

Conference
EAN 2019
Trial
Cohort study, STRIDE, CINRG
Adding ataluren to standard of care slowed disease progression in Duchenne muscular dystrophy patients with nonsense mutation (nmDMD) [1]. This comparison could be made by using data from Strategic Targeting of Registries and International Database of Excellence (STRIDE; n=187) and from the Cooperative International Neuromuscular Research Group (CINRG; n=187) Natural History Study. STRIDE is an observational registry providing data on use of ataluren in nmDMD patients in routine clinical practice. Patients from CINRG, receiving only standard of care, served as controls. Mean age at first symptom onset in the STRIDE and CINRG cohort was 2.7 and 2.9 years, respectively. In both cohorts, the majority of patients received corticosteroids for ≥12 months (56.7% vs 55.1%). In STRIDE, 36 patients (19.3%) lost ambulation compared to 109 (59.2%) in CINRG. This occurred at a later median age in STRIDE: 14.5 versus 11.0 years. Ataluren plus standard of care was...


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