ミグルスタット

危険有害性情報のコード(GHS)

• 絵表示(GHS)

  

• 注意喚起語

  Warning

• 危険有害性情報

  H315：皮膚刺激

  H319：強い眼刺激

• 注意書き

  P264：取扱い後は皮膚をよく洗うこと。

  P264：取扱い後は手や顔をよく洗うこと。

  P280：保護手袋/保護衣/保護眼鏡/保護面を着用するこ と。

  P302+P352：皮膚に付着した場合：多量の水と石鹸で洗うこと。

  P305+P351+P338：眼に入った場合：水で数分間注意深く洗うこと。次にコ ンタクトレンズを着用していて容易に外せる場合は外す こと。その後も洗浄を続けること。

  P332+P313：皮膚刺激が生じた場合：医師の診断/手当てを受けるこ と。

  P337+P313：眼の刺激が続く場合：医師の診断/手当てを受けること。

ミグルスタット 価格

ミグルスタット 化学特性,用途語,生産方法

• 外観

  白色～わずかにうすい褐色, 結晶～結晶性粉末

• 溶解性

  水に溶ける。

• 用途

  糖たん白質糖鎖合成過程、生物機能の解析ツール。

• 効能

  ライソゾーム病治療薬, グルコシルセラミド合成阻害薬

• 商品名

  ブレーザベス (アクテリオンファーマシューティカルズジャパン)

• 説明

  Miglustat is an N-alkylated iminosugar, launched as an oral treatment for mild to moderate type 1 Gaucher’s disease in adult patients for whom enzyme replacement therapy is not a therapeutic option. It is readily synthesized from D-glucose in three steps by first converting to N-butylglucamine via reductive amination with butylamine, followed by a microbial oxidation to an aminofuranose intermediate and subsequent reductive cyclization. Type 1 Gaucher’s disease is a metabolic disorder caused by the lysosomal accumulation of certain glycosphingolipids (GSLs) as a result of deficiency in their degradation. Enlargement of the liver and spleen, low blood platelet and bone lesions are among the key symptoms of this disease. Miglustat acts by inhibiting glucosylceramide synthase, a glucosyl transferase enzyme in the biosynthesis of most GSLs, which results in the lowering of GSLs to a level that can be effectively cleared. Up to 50% reduction in liver and splenocyte GSL levels are achieved in mice by long-term administration of Miglustat (600– 1800 mg/kg/day for 118 days). Miglustat, dosed at 50 and 100 mg in Gaucher patients, exhibits dose proportionate pharmacokinetics (t_max=2.5 h, t_1/2=6 to 7 h) and ＞90% oral bioavailability. Steady-state plasma levels are reached after 4–6 weeks of treatment. Miglustat is not significantly metabolized in humans and the major route of excretion is renal. In clinical trials, efficacy was demonstrated by significant reductions in liver and spleen volumes (12 and 19%, respectively) at 12 months and increase in hemoglobin and platelet count (0.91 g/dL and 13.6×10⁹/l, respectively) at 24 months. Miglustat is generally well tolerated by patients and the most common side effects are diarrhea and weight loss.

• 使用

  Treatment of glycolipid storage diseases.

• 定義

  ChEBI: A hydroxypiperidine that is deoxynojirimycin in which the amino hydrogen is replaced by a butyl group.

• 適応症

  N-butyldeoxynojirimycin is an approved drug for Niemann–Pick disease type C and Gaucher disease type 1, which are lipid storage disorders[1].

• 一般的な説明

  N-Butyldeoxynojirimycin is an alkylated product of imino sugar deoxynojirimycin.

• 生物活性

  Orally active α -glucosidase I and II and ceramide-specific glycosyltransferase inhibitor. Rescues trafficking-deficient F508del-CTFR in human airway epithelial cells via inhibition of ER α -glucosidases I and II. Also has broad spectrum antiviral activity.

ミグルスタット 生産企業

Global(138)Suppliers

ミグルスタット スペクトルデータ(MS)

72599-27-0, ミグルスタット キーワード