Key result
In a real-world registry of 213 boys with nonsense mutation Duchenne muscular dystrophy, the mean age at ataluren treatment start was 9.8 years and mean exposure was 639 days.
Why the study?
The study was designed to provide real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy in routine clinical practice.
Observational (n=213)
Yes
The STRIDE registry provides the largest real-world demographic baseline for boys with nonsense mutation Duchenne muscular dystrophy treated with ataluren.
Answers are grounded in this paper and cite their sources.
Real-world patterns of ataluren initiation in nonsense-mutation DMD inform timing considerations; leaves open efficacy and outcome questions.
Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population. Patients & methods: Patients will be followed up from enrollment for ≥5 years or until study withdrawal. Results & conclusion: As of 9 July 2018, 213 DMD boys were enrolled from 11 countries. Mean (standard deviation) ages at first symptoms and at study treatment start were 2.7 (1.7) years and 9.8 (3.7) years, respectively. Corticosteroids were used by 190 patients (89.2%) before data cut-off. Mean (standard deviation) ataluren exposure was 639.0 (362.9) days. Six patients withdrew. STRIDE is the first drug registry for patients with DMD and represents the largest real-world registry of patients with nmDMD to date.
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Muntoni et al. (2019) conducted an observational in Nonsense mutation Duchenne muscular dystrophy (n=213). Ataluren was evaluated on Initial demographic characteristics and ataluren exposure. In a real-world registry of 213 boys with nonsense mutation Duchenne muscular dystrophy, the mean age at ataluren treatment start was 9.8 years and mean exposure was 639 days.
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