To the top

Page Manager: Webmaster
Last update: 9/11/2012 3:13 PM

Tell a friend about this page
Print version

Ataluren use in patients … - University of Gothenburg, Sweden Till startsida
Sitemap
To content Read more about how we use cookies on gu.se

Contact form








 


Note! If you want an answer on a question you must specify your email address




Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

Journal article
Authors F. Muntoni
I. Desguerre
M. Guglieri
A. N. Osorio
J. Kirschner
Mar Tulinius
F. Buccella
G. Elfring
C. Werner
T. Schilling
P. Trifillis
O. Zhang
A. Delage
C. L. Santos
E. Mercuri
Published in Journal of Comparative Effectiveness Research
Volume 8
Issue 14
Pages 1187-1200
ISSN 2042-6305
Publication year 2019
Published at Institute of Clinical Sciences, Department of Pediatrics
Pages 1187-1200
Language en
Links dx.doi.org/10.2217/cer-2019-0086
Subject categories Clinical Medicine

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.

Page Manager: Webmaster|Last update: 9/11/2012
Share:

The University of Gothenburg uses cookies to provide you with the best possible user experience. By continuing on this website, you approve of our use of cookies.  What are cookies?