Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry (P7-6.008)

20250 citationsJournal Article

Authors

Christian Werner

Eugenio Mercuri · Catholic University of America

Filippo Buccella · Parent Project Onlus

A. Nascimento Osorio · Hospital Sant Joan de Déu Barcelona

M. Tulinius · University of Gothenburg

Isabelle Desguerre · Hôpital Necker-Enfants Malades

Maria Bernadete Dutra de Resende · Universidade de São Paulo

Abstract

To assess the reliability of ataluren effectiveness data in Strategic Targeting of Registries and International Database of Excellence (STRIDE [NCT02369731]) nonsense mutation DMD (nmDMD) patients and whether mutation type is a source of bias.

Topics & Keywords

Lanthanide and Transition Metal Complexes Biomedical Research and Pathophysiology Amino Acid Enzymes and Metabolism

Publication Details

Published in: Neurology

Volume 104, Issue 7_Supplement_1

DOI: 10.1212/wnl.0000000000211740

Field-Weighted Citation Impact: 0.00

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Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry (P7-6.008)

Authors

Abstract

Topics & Keywords

Publication Details