Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials
Mario Cortina-Borja
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Danielle te Vruchte
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Eugen Mengel
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Yasmin Amraoui
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Jackie Imrie
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...
Lydia Aston
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Rachel Shaw
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Rebecca Knibb
S Tay
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X He
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AM Jenner
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BS Wong
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WY Ong
Ting-Ting Chu
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Xintao Tu
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Kun Yang
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Jianjun Wu
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Joyce J. Repa
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...
2020 ◽
Vol 15
(1)
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pp. e0227829
Niamh X. Cawley
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Caitlin Sojka
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Antony Cougnoux
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Anna T. Lyons
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Elena‐Raluca Nicoli
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...