Mercuri, Eugenio
Osorio, Andrés Nascimento
Muntoni, Francesco http://orcid.org/0000-0002-9102-5232
Buccella, Filippo
Desguerre, Isabelle
Kirschner, Janbernd
Tulinius, Már
de Resende, Maria Bernadete Dutra
Morgenroth, Lauren P.
Gordish-Dressman, Heather http://orcid.org/0000-0002-2330-5427
Johnson, Shelley
Kristensen, Allan
Werner, Christian http://orcid.org/0000-0002-6139-7473
Trifillis, Panayiota
Henricson, Erik K.
McDonald, Craig M.
,
Clinical trials referenced in this document:
Documents that mention this clinical trial
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
https://doi.org/10.1007/s00415-023-11687-1
Funding for this research was provided by:
PTC Therapeutics
Università Cattolica del Sacro Cuore
Article History
Received: 15 November 2022
Revised: 24 March 2023
Accepted: 26 March 2023
First Online: 28 April 2023
Change Date: 17 July 2023
Change Type: Correction
Change Details: A Correction to this paper has been published:
Change Details: https://doi.org/10.1007/s00415-023-11864-2
Declarations
:
: EM has acted as an advisory board member for AveXis, Biogen, BioMarin, Bristol-Myers Squibb, Ionis Pharmaceuticals, Italfarmaco, Prosensa, PTC Therapeutics, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics and Summit Therapeutics. ANO has received speaker and consultancy fees from PTC Therapeutics and is an investigator on clinical trials sponsored by Biogen, Italfarmaco, Roche, Sarepta Therapeutics and TAMDMD. FM has received consultancy fees from Akashi Therapeutics, Biogen, BioMarin, Catabasis, Italfarmaco, Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics and Tivorsan Pharmaceuticals, and is supported by University College London, London, UK, and the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children, National Health Service Foundation Trust. FB has received consultancy fees from PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. ID has received consultancy fees from AveXis, Biogen, BioMarin and PTC Therapeutics. JK has acted as a consultant for Biogen, Novartis, Pfizer, PTC Therapeutics, Roche and Scholar Rock and has received research support for taking part in clinical research from Biogen, Novartis, PTC Therapeutics and Roche. MT has received lecture fees from Biogen and PTC Therapeutics; has acted as a consultant on DMD clinical trials for BioMarin, PTC Therapeutics, ReveraGen BioPharma and Sarepta Therapeutics; and has acted as an advisory board member for AveXis, Biogen and PTC Therapeutics. LM is a part owner of TRiNDS. HGD has served as a consultant for AGADA Biosciences, ReveraGen BioPharma and Solid GT, and is a cofounder and part owner of TRiNDS. SJ, AK and PT are full-time employees of PTC Therapeutics, Inc. CW is a full-time employee of PTC Therapeutics GmbH. EH has acted as a consultant on clinical trials of DMD for Capricor Therapeutics, Epirium Bio (formerly Cardero Therapeutics), Mallinckrodt Pharmaceuticals, Pfizer, PTC Therapeutics, Sanofi Genzyme, Santhera Pharmaceuticals and Sarepta Therapeutics. CM has served as a consultant for clinical trials with Astellas Pharma, Avidity Biosciences, BioMarin Pharmaceutical, Capricor Therapeutics, Catabasis Pharmaceuticals, Edgewise Therapeutics, Entrada Therapeutics, Epirium Bio (formerly Cardero Therapeutics), FibroGen, Hoffman La Roche, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals Sarepta Therapeutics and Solid Biosciences. He has received research support for clinical trials from Capricor Therapeutics, Catabasis Pharmaceuticals, Edgewise Therapeutics, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. He serves on external advisory boards related to DMD for BioMarin Pharmaceutical, Capricor Therapeutics, Edgewise Therapeutics, Eli Lilly, PTC Therapeutics, Sarepta Therapeutics and Santhera Pharmaceuticals. MBDR reports no disclosures of interest.
: Before any patient is enrolled and study-related data are collected, ethics committee approval of the protocol, informed consent form and all patient enrollment materials are obtained in each country and for each site, as applicable. The study is conducted in accordance with the ethical principles outlined in the Declaration of Helsinki, applicable privacy laws and local regulations for each participating site, as well as with the Guidelines for Good Pharmacoepidemiology Practices.