Migalastat
Drug Details
- Description
- Migalastat is an oral pharmacological chaperone used to treat Fabry disease. It stabilizes certain mutant forms of the enzyme alpha-galactosidase A, improving its function and reducing the buildup of globotriaosylceramide (GL-3).
- Alternative Names
- Migalastat is the generic name. The brand name is Galafold.
- Key Genes
- Migalastat targets the GLA gene, which encodes the enzyme alpha-galactosidase A. This is relevant for treating Fabry disease, caused by mutations in the GLA gene.
- Drug Use
- Migalastat is used to treat Fabry disease, a genetic disorder that leads to the buildup of a particular type of fat in the body, causing symptoms such as pain, kidney failure, heart issues, and stroke. It works by stabilizing certain enzyme mutations to help the body break down the accumulated fat.
- Genetic Factors Influencing Response
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