Muntoni, F;
Desguerre, I;
Guglieri, M;
Osorio, AN;
Kirschner, J;
Tulinius, M;
Buccella, F;
... Mercuri, E; + view all
(2019)
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.
Journal of Comparative Effectiveness Research
10.2217/cer-2019-0086.
(In press).
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Abstract
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.
| Type: | Article |
|---|---|
| Title: | Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.2217/cer-2019-0086 |
| Publisher version: | https://doi.org/10.2217/cer-2019-0086 |
| Language: | English |
| Additional information: | © 2019 Future Science Group. This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
| Keywords: | STRIDE Registry, ataluren, demographics, dystrophin, nonsense mutation Duchenne muscular dystrophy |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10080544 |
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