%0 Journal Article
%T Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review
%A Christian Velten
%A Jose Luis Insua
%A Katar¨ªna Jur¨ª£¿kov¨¢
%A Mercedes Pineda
%A Miriam Kolnikova
%A Parvaneh Karimzadeh
%A Stefan A. Kolb
%A Vera Malinova
%J Archive of "Orphanet Journal of Rare Diseases".
%D 2019
%R 10.1186/s13023-019-0996-6
%X The online version of this article (10.1186/s13023-019-0996-6) contains supplementary material, which is available to authorized users
%K Niemann-pick disease type C
%K NP-C
%K Disease characteristics
%K Prognosis
%K NP-C disability scales
%U https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6367842/