Dermatologic Manifestations of Niemann-Pick Disease Medication

Updated: Apr 12, 2021
  • Author: Robert A Schwartz, MD, MPH; Chief Editor: Dirk M Elston, MD  more...
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Medication Summary

Miglustat, an inhibitor of glycosphingolipid synthesis, may have some efficacy. A 24-month course of treatment in Niemann-Pick disease type C patients was reported to result in mild clinical improvement or stabilization. These preliminary results require confirmation. [31] Long-term miglustat therapy seems to stabilize neurological disease and be well tolerated in adult and juvenile patients with Niemann-Pick disease type C. [32]