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[转载]Fabry Disease: Developing Drugs for Treatment Guidance for 2024-07-09
I.    INTRODUCTION The purpose of this guidance is to provide recommendations to sponsors regarding clinical trial design features ...
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[转载]EMA: NOTIFICATION OF AMENDMENTS AND RELATED MEASUR 2024-06-20
3.  NOTIFICATION   OF    AMENDMENTS   AND    RELATED MEASURES 3.1.  Legal basis and s ...
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[转载]Management of Adults and Children receiving CAR T-cell thera 2024-06-11
ABSTRACT: Background: Several commercial and academic autologous chimeric antigen receptor T-cell products targeting ...
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[转载]Onasemnogene abeparvovec基因疗法治疗症状性婴儿型脊髓性肌萎缩症1型-part2 2024-05-21
Onasemnogene abeparvovec 基因疗法治疗症状性婴儿型脊髓性肌萎缩症 1 型   Summary  摘要 Background Spinal muscular atrophy is a r ...
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[转载]Onasemnogene abeparvovec基因疗法治疗症状性婴儿型脊髓性肌萎缩症1型-part1 2024-05-21
Onasemnogene abeparvovec 基因疗法治疗症状性婴儿型脊髓性肌萎缩症 1 型   Summary  摘要 Background Spinal muscular atrophy type ...
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[转载]治疗 SMA 罕见病的孤儿药 Onasemnogene Abeparvovec (Zolgensma ® ) 的真实世界 2024-05-21
治疗 SMA 罕见病的孤儿药 Onasemnogene Abeparvovec (Zolgensma ® ) 的真实世界安全性数据:基于 EMA 不良事件报告系统的药物警戒研究   Abst ...
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[转载]JEWELFISH 24月结果 2024-05-21
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam   A ...
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