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

Year: 2025

Type: article

Source: Neurology

DOI: 10.1212/wnl.0000000000211740

Authors: Christian Werner, Eugenio Mercuri, Filippo Buccella, Andrés Nascimento Osorio, Már Tulinius, Isabelle Desguerre, Maria Bernadete Dutra de Resende, Craig McDonald, Heather Gordish-Dressman, Lauren Morgenroth, Shelley Johnson, Alexis Krolick, Balaji Anbu, Emelline Liu, Francesco Muntoni

Institutions: Università Cattolica del Sacro Cuore, Pontifícia Universidade Católica de São Paulo, Hospital Sant Joan de Déu Barcelona, Universitat de Barcelona, Drottning Silvias barn- och ungdomssjukhus, University of Gothenburg, Hôpital Necker-Enfants Malades, Universidade de São Paulo, University of California, Davis, Children's National, PTC Therapeutics (United States), University College London

Language: English

Topic: Muscle Physiology and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4409227654

FWCI:

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Related to: 10