Cơ Chế Tác Dụng :
Fx-1006A is a small molecule compound with the potential to treat genetic disorders, such as familial amyloid cardiomyopathy (FAC) and familial amyloid polyneuropathy (FAP).
Fx-1006A is a first-in-class, disease-modifying compound that is designed to inhibit the formation of amyloid deposits by preventing the misfolding and deposition of the transthyretin protein (TTR), which is associated with amyloidosis.
Chỉ Định :
Investigated for use/treatment in amyloidosis.