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Efficacy and Safety Data (52-Week) from a Phase 1/2 Trial and Extension Study of JR-171 (lepunafusp Alfa) Used in Enzyme Replacement Therapy for Patients with MPS I

Roberto Giugliani,Paul Harmatz,Ana Maria Martins,Takashi Hamazaki,Ryutaro Kira,Toru Kubo,Yuji Sato,Hiroaki Moriuchi,Satoshi Kawashima,Toshiaki Ikeda,Sairei So,Kazunori Tanizawa,Mathias Schmidt

Molecular Genetics and Metabolism（2024）

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关键词
Enzyme Replacement Therapy
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