Delayed pulmonary progression in Golodirsen-treated patients with Duchenne muscular dystrophy vs mutation-matched external controls

J. Iff, E. Tuttle,Y. Liu, F. Wei, N. Done,L. Servais, A. Seferian, V. Straub,M. Guglieri,E. Mercuri,F. Muntoni

NEUROMUSCULAR DISORDERS(2023)

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关键词
pulmonary progression,muscular dystrophy,duchenne,golodirsen-treated,mutation-matched
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