Journal article

Miglustat in Niemann-Pick disease type C patients: a review

Merce Pineda, Mark Walterfang, Marc C Patterson

Orphanet Journal of Rare Diseases | BMC | Published : 2018

Abstract

OBJECTIVE: Niemann-Pick disease type C (NP-C) is a rare, autosomal recessive, neurodegenerative disease associated with a wide variety of progressive neurological manifestations. Miglustat is indicated for the treatment of progressive neurological manifestations in both adults and children. Since approval in 2009 there has been a vast growth in clinical experience with miglustat. The effectiveness of miglustat has been assessed using a range of measures. METHODS: Comprehensive review of published data from studies of cellular neuropathological markers and structural neurological indices in the brain, clinical impairment/disability, specific clinical neurological manifestations, and patient s..

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University of Melbourne Researchers