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Impact of imiglucerase supply shortage on clinical and laboratory parameters in Norrbottnian patients with Gaucher disease type 3.
Arch Immunol Ther Exp (Warsz). 2015 Feb;63(1):65-71. doi: 10.1007/s00005-014-0308-8. Epub 2014 Sep 10.
Arch Immunol Ther Exp (Warsz). 2015.
PMID: 25205209
Free PMC article.
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.
Patterson MC, Mengel E, Vanier MT, Schwierin B, Muller A, Cornelisse P, Pineda M; NPC Registry investigators.
Patterson MC, et al.
Orphanet J Rare Dis. 2015 May 28;10:65. doi: 10.1186/s13023-015-0284-z.
Orphanet J Rare Dis. 2015.
PMID: 26017010
Free PMC article.
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