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
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..View full abstract
Matthew Reilly PhD at InTouch Medical Ltd. provided editorial support in the preparation of this manuscript for publication, paid for by Actelion, a Janssen Pharmaceutical Company of Johnson and Johnson.