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

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 survival.

Results

Cranial diffusion tensor imaging and magnetic resonance spectro

SUBMITTER: Pineda M 

PROVIDER: S-EPMC6094874 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature