The following new article has just been published in Orphanet Journal of Rare Diseases
For articles using Author Version-first publication you will see a provisional PDF corresponding to the accepted manuscript. In these instances, the fully formatted Final Version PDF and full text (HTML) versions will follow in due course.
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort studyPatterson MC, Mengel E, Vanier MT, Schwierin B, Muller A, Cornelisse P, Pineda M, On behalf of the NPC Registry investigators
Orphanet Journal of Rare Diseases 2015, 10 :65 (28 May 2015)