Cortina-Borja, M;
Te Vruchte, D;
Mengel, E;
Amraoui, Y;
Imrie, J;
Jones, SA;
I Dali, C;
... Platt, FM; + view all
(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.