Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis

202344 citationsJournal Articlehybrid Open Access

Authors

Eugenio Mercuri · Università Cattolica del Sacro Cuore

A. Nascimento Osorio · Instituto de Salud Carlos III

Francesco Muntoni · Great Ormond Street Hospital

Filippo Buccella · Parent Project Onlus

Isabelle Desguerre · Hôpital Necker-Enfants Malades

Janbernd Kirschner · University of Freiburg

M. Tulinius

Abstract

Long-term, real-world treatment with ataluren plus SoC delays several disease progression milestones in individuals with nmDMD. NCT02369731; registration date: February 24, 2015.

Topics & Keywords

Muscle Physiology and Disorders Cardiomyopathy and Myosin Studies Genetic Neurodegenerative Diseases

UN Sustainable Development Goals

Partnerships for the goals

Publication Details

Published in: Journal of Neurology

Volume 270, Issue 8, pp. 3896-3913

DOI: 10.1007/s00415-023-11687-1

Field-Weighted Citation Impact: 6.78