눈에 묻으면 몇 분간 물로 조심해서 씻으시오. 가능하면 콘택트렌즈를 제거하시오. 계속 씻으시오.
NFPA 704
0
2
0
DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE C화학적 특성, 용도, 생산
개요
Migalastat,
which is marketed by Amicus Therapeutics, received approval
in the EU for the treatment of Fabry disease in adults and
adolescents aged 16 or older. Fabry disease is caused by
mutations of the enzyme α-galactosidase A (α-GAL A) that
cause protein misfolding and prevents efficient metabolism of
the glycosphingolipid globotriaosylceramide (GL3). Accumulation
of GL3 in lysosomes, blood vessels, and various tissues
ultimately leads to significant heart, kidney, and dermatological
problems. Migalastat functions as a molecular chaperone to α-
GAL A, engaging the enzyme and enabling it to adopt the
proper conformation allowing for efficient breakdown of
GL3. Because the standard of care prior to 2016 for treating
Fabry disease was enzyme replacement therapy (ERT),
migalastat’s approval in the EU represents an important
advance for patients suffering from this disorder.
화학적 성질
White Crystalline Solid
용도
Proven to be an extremely potent and selective a-D-galactosidase inhibitor.
DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE 준비 용품 및 원자재
