User:Mr. Ibrahem/Miglustat

Miglustat, sold under the brand name Zavesca, is a medication used to treat Gaucher disease type 1 (GD1) and Niemann–Pick disease type C (NPC). It is taken by mouth.

Common side effects include weight loss, shaking, diarrhea, increased intestinal gas, and abdominal pain. Other side effects may include peripheral neuropathy and low platelets. Safety in pregnancy is unclear. It is a glucosylceramide synthase inhibitor.

Miglustat was approved for medical use Europe in 2002 and the United States in 2003. It is available as a generic medication. In the United Kingdom 4 weeks of medication costs the NHS about £3,400 as of 2021. This amount in the United States costs about 23,000 USD.