Miglustat
From Wikipedia, the free encyclopedia
 |
|
|
Miglustat
|
|
| Systematic (IUPAC) name | |
| 1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol | |
| Identifiers | |
| CAS number | |
| ATC code | A16 |
| PubChem | |
| DrugBank | |
| Chemical data | |
| Formula | C10H21NO4 |
| Mol. mass | 219.28 g/mol |
| Pharmacokinetic data | |
| Bioavailability | 97% |
| Protein binding | Nil |
| Metabolism | Nil |
| Half life | 6–7 hours |
| Excretion | Renal, unchanged |
| Therapeutic considerations | |
| Licence data |
|
| Pregnancy cat. |
X(US) |
| Legal status | |
| Routes | Oral |
Miglustat is a drug used to treat Gaucher disease.
It inhibits the enzyme glucosylceramide synthase,[1] an essential enzyme for the synthesis of most glycosphingolipids.
It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. [2]
It is marketed under the trade name Zavesca.
It has also been investigated for use in treating Niemann-Pick disease.[3]
[edit] References
- ^ van Giersbergen PL, Dingemanse J (2007). "Influence of Food Intake on the Pharmacokinetics of Miglustat, an Inhibitor of Glucosylceramide Synthase". doi:. PMID 17720777.
- ^ Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P (2005). "Guidance on the use of miglustat for treating patients with type 1 Gaucher disease". Am. J. Hematol. 80 (3): 223–9. doi:. PMID 16247743.
- ^ Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE (2007). "Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study". Lancet neurology 6 (9): 765–72. doi:. PMID 17689147.
 |
This pharmacology-related article is a stub. You can help Wikipedia by expanding it. |
|
|||||||||||
