Tìm theo
Migalastat
Thuốc Gốc
Small Molecule
Amigal (migalastat hydrochloride) is an experimental, oral therapy for the treatment of Fabry disease and belongs to a class of molecules known as pharmacological chaperones.
Cơ Chế Tác Dụng : Amigal (migalastat hydrochloride) is an experimental, oral therapy for the treatment of Fabry disease and belongs to a class of molecules known as pharmacological chaperones. Amigal acts by selectively binding to the misfolded enzyme responsible for Fabry disease, α-GAL. This may increase the enzyme’s stability and promote the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced and the enzyme can perform its normal biological function, which is the breakdown of its natural substrate, GL-3.
Chỉ Định : Investigated for use/treatment in fabry disease.
Dữ Kiện Thương Mại
Nhà Sản Xuất
  • Công ty :
    Sản phẩm biệt dược : Amigal
Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB05018
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