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

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 ...

Source: Orphanet Journal of Rare Diseases - August 15, 2018 Category: Internal Medicine Authors: Merc è Pineda, Mark Walterfang and Marc C. Patterson Tags: Review Source Type: research