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pro vyhledávání: '"Robert Giugliani"'
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
Publikováno v:
Journal of Inherited Metabolic Disease
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
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::89ee288721a12870f8e8b8e36c245ac0
https://doi.org/10.1002/jimd.12245
https://doi.org/10.1002/jimd.12245
