Long‐term survival outcomes of patients with Niemann‐Pick disease type C receiving miglustat treatment: A large retrospective observational study

Autor:	Bénédicte Héron, Marie T. Vanier, Marc C. Patterson, Robert Giugliani, Daniel W. Rosenberg, William S. Garver, Jackie Imrie, Olivier Morand, Yann Nadjar, Patrick Moneuse, F. John Meaney, Barbara Schwierin, Helena Jahnová
Rok vydání:	2020
Předmět:	medicine.medical_specialty NP‐C Zavesca NPC registry Disease survival 03 medical and health sciences observational national cohorts Niemann‐pick disease type C Internal medicine Miglustat Genetics Risk of mortality Medicine Genetics (clinical) 030304 developmental biology 0303 health sciences Niemann–Pick disease type C business.industry Proportional hazards model 030305 genetics & heredity Hazard ratio Retrospective cohort study Original Articles medicine.disease Original Article miglustat Observational study business medicine.drug
Zdroj:	Journal of Inherited Metabolic Disease
ISSN:	1573-2665 0141-8955
Popis:	Miglustat has been indicated for the treatment of Niemann‐Pick disease type C (NP‐C) since 2009. The aim of this observational study was to assess the effect of miglustat on long‐term survival of patients with NP‐C. Data for 789 patients from five large national cohorts and from the NPC Registry were collected and combined. Miglustat‐treated and untreated patients overall and within sub‐groups according to age‐at‐neurological‐onset, that is, early infantile‐onset (
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::89ee288721a12870f8e8b8e36c245ac0 https://doi.org/10.1002/jimd.12245 Zobrazit plný text záznamu Plný text