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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

✍ Scribed by Marc C Patterson; Darleen Vecchio; Helena Prady; Larry Abel; James E Wraith

Book ID: 114408693
Publisher: The Lancet
Year: 2007
Tongue: English
Weight: 150 KB
Volume: 6
Category: Article
ISSN: 1474-4465
DOI: 10.1016/s1474-4422(07)70194-1

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## Abstract Niemann–Pick disease type C (NPC) is a progressive neurodegenerative disorder for which there is no effective treatment other than supportive therapy. Recently, the oral medication miglustat has been offered as a possible therapy aimed at reducing pathological substrate accumulation. Th