Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Cortina-Borja, M; Te Vruchte, D; Mengel, E; Amraoui, Y; Imrie, J; Jones, SA; I Dali, C; ... Platt, FM; + view all Cortina-Borja, M; Te Vruchte, D; Mengel, E; Amraoui, Y; Imrie, J; Jones, SA; I Dali, C; Fineran, P; Kirkegaard, T; Runz, H; Lachmann, R; Bremova-Ertl, T; Strupp, M; Platt, FM; - view fewer (2018) Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials. Orphanet Journal of Rare Diseases , 13 (1) , Article 143. 10.1186/s13023-018-0880-9 . Green open access

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