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  • × Orphanet Journal of Rare Disease
 全选  【符合条件的数据共：3040条】

1 Toward patient-centered treatment goals for duchenne muscular dystrophy: insights from the “Your Voice” study [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Carolyn E. Schwartz;Skyler Jackson;Patti Engel;等

关键词:Duchenne muscular dystrophy;Treatment;...

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2 Randomized controlled trial data for successful new drug application for rare diseases in the United States [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Yosuke Kubota, Mamoru Narukawa

关键词:Rare diseases;Orphan drugs;...

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3 Association between one-session bilateral whole-lung lavage and periprocedural complications in patients with pulmonary alveolar proteinosis: a retrospective cohort study [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Yuelun Zhang;Xiaohan Xu;Yuguang Huang;等

关键词:Whole-lung lavage;One-session;...

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4 A global neuronopathic gaucher disease registry (GARDIAN): a patient-led initiative [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Elin Haf Davies;Kathleen Beusterien;Dena H. Jaffe;等

关键词:Registry;Rare disease;...

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5 Patient-reported outcomes in Gaucher’s disease: a systematic review [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Yue Wang;Junchao Feng;Shunping Li;等

关键词:Quality of life;Patient-reported outcomes;...

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6 How to START? Four pillars to optimally begin your orphan drug development [期刊论文]

来源：Orphanet Journal of Rare Diseases , 2023

作者:Liliana Batista;Diego Ardigo;Virginie Hivert;等

关键词:Orphan drugs;Drug development;...

使用许可:CC BY

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