GALAFOLD (migalastat HCl)
METABOLIC DISEASE ENZYME REPLACEMENT, FABRY'S DISEASE · FABRY DISEASE
AMICUS THERAPEU
Drug class
Pharmacological Chaperone - Alpha-galactosidase A stabilizer
The class of pharmacological chaperones that stabilize alpha-galactosidase A consists of small-molecule agents designed to correct the misfolding of deficient lysosomal enzymes. Read more about Pharmacological Chaperone - Alpha-galactosidase A stabilizer
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The class of pharmacological chaperones that stabilize alpha-galactosidase A consists of small-molecule agents designed to correct the misfolding of deficient lysosomal enzymes.
Agents in this class reversibly bind to the active site of specific amenable mutant forms of alpha-galactosidase A, promoting proper folding, facilitating trafficking from the endoplasmic reticulum to the lysosomes, and preserving catalytic activity.
Therapeutically, this mechanism enables the breakdown of accumulated glycosphingolipids, primarily globotriaosylceramide, and is indicated for the management of Fabry disease in patients harboring responsive genetic variants.
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patsnap.com
The class of pharmacological chaperones that stabilize alpha-galactosidase A consists of small-molecule agents designed to correct the misfolding of deficient lysosomal enzymes Agents in this class reversibly bind to the active site of specific amenable mutant forms of alpha-galactosidase A, promoting proper folding, facilitating trafficking from the endoplasmic reticulum to the lysosomes, and preserving catalytic activity Therapeutically, this mechanism enables the breakdown of accumulated glyc...
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mdpi.com
The class of pharmacological chaperones that stabilize alpha-galactosidase A consists of small-molecule agents designed to correct the misfolding of deficient lysosomal enzymes
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fepblue.org
Agents in this class reversibly bind to the active site of specific amenable mutant forms of alpha-galactosidase A, promoting proper folding, facilitating trafficking from the endoplasmic reticulum to the lysosomes, and preserving catalytic activity Therapeutically, this mechanism enables the breakdown of accumulated glycosphingolipids, primarily globotriaosylceramide, and is indicated for the management of Fabry disease in patients harboring responsive genetic variants
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