Neuropediatrics 2025; 56(S 01): S1-S24
DOI: 10.1055/s-0045-1812120
DOI: 10.1055/s-0045-1812120
Neuromuscular Disorders
Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry
Authors
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C. Werner
1 PTC Therapeutics Germany GmbH, Frankfurt am Main, Germany -
J. Gruenert
1 PTC Therapeutics Germany GmbH, Frankfurt am Main, Germany -
E. Mercuri
2 Department of Pediatric Neurology, Catholic University, Rome, Italy -
F. Buccella
3 Parent Project APS Italy, Rome, Italy -
A. N. Osorio
4 Hospital Sant Joan de Déu, Universidad de Barcelona, Unidad de Patología Neuromuscular, Barcelona, Spain -
M. Tulinius
5 Department of Pediatrics, Queen Silvia Children's Hospital, University of Gothenburg, Gothenburg, Sweden -
I. Desguerre
6 Hôpital Necker, Enfants Malades, Paris, France -
M. B. Dutra de Resende
7 Department of Neurology, Faculty of Medicine, University of São Paulo, São Paulo, Brazil -
C. McDonald
8 University of California Davis School of Medicine, Davis, California, United States -
H. Gordish-Dressman
9 Center for Genetic Medicine, Children's National Health System and the George Washington University, Washington, District of Columbia, United States -
L. Morgenroth
10 Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), Pittsburgh, Pennsylvania, United States -
S. Johnson
11 PTC Therapeutics Inc., Warren, New Jersey, United States -
A. Krolick
11 PTC Therapeutics Inc., Warren, New Jersey, United States -
B. Anbu
11 PTC Therapeutics Inc., Warren, New Jersey, United States -
E. Liu
11 PTC Therapeutics Inc., Warren, New Jersey, United States -
F. Muntoni
12 University College London Great Ormond Street Institute of Child Health, London, United Kingdom
