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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M. Strupp | H. Runz | R. Lachmann | M. Cortina-Borja | F. Platt | T. Kirkegaard | E. Mengel | Danielle te Vruchte | J. Imrie | T. Bremova-Ertl | C. í Dali | Simon A. Jones | Yasmina Amraoui | Simon A Jones | P. Fineran