Ingredient

Migalastat Hydrochloride

Miglustat hydrochloride is an ingredient that assists enzymatic activity for the treatment of Fabry disease.

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Prescription medication
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MFDS ingredient code M267107

What does it do?

Miglustat hydrochloride is a drug ingredient used for the treatment of Fabry disease, a rare genetic disorder. This ingredient improves enzyme function and helps alleviate metabolic disorders. It mainly acts as an enzymatic activity enhancer.

Miglustat hydrochloride binds to the defective alpha-galactosidase A enzyme, stabilizing its structure. As a result, the enzyme can function properly, promoting the breakdown of abnormal substances accumulated in the body. Consequently, it helps alleviate symptoms of Fabry disease.

Commonly used for

Approved uses differ by product. Check each product's information for its actual use.

Good to know

Unlike enzyme replacement therapy, miglustat hydrochloride can be taken orally and has a mechanism that directly assists enzymatic activity. This is a distinguishing feature from existing therapies, known to be a personalized treatment that responds to specific genetic mutations.

Medicines containing this ingredient

Even with the same ingredient, strength, dosage form and how it is taken can differ.

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Dosage form
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1 total

Even with the same ingredient, dosage and approved uses can differ by strength, dosage form and combined ingredients. Check each product's information before taking it.

Questions about this ingredient

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For anything that needs medical judgment, consult a doctor or pharmacist.

Product approval information is based on drug information provided by the Ministry of Food and Drug Safety.
Ingredient and class descriptions are not part of MFDS data and were written separately by Cashdoc.
Consult a doctor or pharmacist for accurate dosage, administration, and usage instructions.