Neurology Central

Ataluren use in nonsense mutation Duchenne muscular dystrophy: patient information from the STRIDE Registry

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Lay abstract

Duchenne muscular dystrophy (DMD) is a rare disorder that causes progressive muscle degeneration and weakness. In around 10–15% of boys with DMD, the disorder is caused by a nonsense mutation in the DMD gene, so-called nonsense mutation (nm)DMD. Ataluren is a first-in-class, orally bioavailable treatment for patients with nmDMD. The STRIDE Registry is an ongoing, multicenter, observational, post-approval safety study of ataluren in patients with nmDMD, which was set up to gather data on patterns of use and long-term outcomes in real-world clinical practice. The STRIDE Registry is the first drug registry for DMD patients and represents the largest real-world registry of nmDMD patients to date. In this paper, the authors describe the initial demographic characteristics of the study population in the registry.

Abstract

Aims: STRIDE is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in nmDMD patients 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 & Conclusions: As of 9 July 2018, 213 DMD boys were enrolled from 11 countries. Mean (SD) 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 (SD) ataluren exposure was 639.0 (362.9) days. Six patients withdrew. STRIDE is the first drug registry for DMD patients and represents the largest real-world registry of nmDMD patients to date.

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