Additional file 1: of Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Cortina-Borja, Mario; te Vruchte, Danielle; Mengel, Eugen; Amraoui, Yasmin; Imrie, Jackie; Jones, Simon; i Dali, Christine; Fineran, Paul; Kirkegaard, Thomas; Runz, Heiko; Lachmann, Robin; Bremova-Ertl, Tatiana; Strupp, Michael; Platt, Frances

doi:10.6084/m9.figshare.6978362.v1

13023_2018_880_MOESM1_ESM.docx (15.62 kB)

Additional file 1: of Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

journal contribution

posted on 2018-08-16, 05:00 authored by Mario Cortina-Borja, Danielle te Vruchte, Eugen Mengel, Yasmin Amraoui, Jackie Imrie, Simon Jones, Christine i Dali, Paul Fineran, Thomas Kirkegaard, Heiko Runz, Robin Lachmann, Tatiana Bremova-Ertl, Michael Strupp, Frances Platt

Table S1. Spearmanâs correlations between the 8 possible subdomains and the total severity score calculated, including or excluding the subdomains in question. (DOCX 15 kb)

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Niemann-Pick disease type C NPC Annual severity increment score ASIS Clinical severity scale Clinical trials Experimental therapy Acetyl-DL-leucine Tanganil

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Additional file 1: of Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

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