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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

Eugenio Mercuri,Andrés Nascimento Osorio,Francesco Muntoni,Filippo Buccella,Isabelle Desguerre,Janbernd Kirschner,Már Tulinius,Maria Bernadete Dutra de Resende,Lauren P. Morgenroth,Heather Gordish-Dressman,Shelley Johnson,Allan Kristensen,Christian Werner,Panayiota Trifillis,Erik K. Henricson,Craig M. McDonald

Journal of Neurology（2023）

Cited 13|Views48

Key words

Ataluren,Effectiveness,Nonsense mutation Duchenne muscular dystrophy,Safety,STRIDE Registry

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