Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry (P7-6.008)
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.
Authors
- Isabelle Desguerre (ORCID: https://orcid.org/0000-0002-4074-5862)
- A. Nascimento Osorio
- Eugenio Maria Mercuri (ORCID: https://orcid.org/0000-0002-9851-5365)
- Shelley Johnson (ORCID: https://orcid.org/0009-0003-1291-4264)
- M. Tulinius
- Enwu Liu (ORCID: https://orcid.org/0000-0003-2580-3523)
- Lauren P. Morgenroth
- Christian Werner (ORCID: https://orcid.org/0000-0002-6139-7473)
- Craig M. McDonald (ORCID: https://orcid.org/0000-0002-8779-3220)
- Filippo Buccella (ORCID: https://orcid.org/0000-0002-1704-0143)
- Heather Gordish‐Dressman (ORCID: https://orcid.org/0000-0002-2330-5427)
- Francesco Muntoni (ORCID: https://orcid.org/0000-0002-9102-5232)
- B. Anbu
- Alexis Krolick
- Maria Bernadete Dutra de Resende
Institutions
- Hôpital Necker-Enfants Malades (FR)
- Hospital Sant Joan de Déu Barcelona (ES)
- Children's National (US)
- Universidade de São Paulo (BR)
- Great Ormond Street Hospital (GB)
- Parent Project Onlus (IT)
- Drottning Silvias barn- och ungdomssjukhus (SE)
- Clinical Research Solutions (US)
- PTC Therapeutics (United States) (US)
- University College London (GB)
- Universitat de Barcelona (ES)
- University of California, Davis (US)
- Catholic University of America (US)
- University of Gothenburg (SE)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211740
- Primary Topic
- Lanthanide and Transition Metal Complexes
- Type
- article
- Field-Weighted Citation Impact
- 0.00