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Galafold
Galafold (miglustat) is an orally administered medication used for the treatment of Fabry disease, a genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A. This medication works by inhibiting the production of globotriaosylceramide (Gb3), which accumulates in various tissues due to the enzyme deficiency. By reducing Gb3 levels, Galafold helps alleviate symptoms and prevent complications associated with Fabry disease.
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Frequently Asked Questions
How long does it take for Galafold to work?
Some patients may notice improvements within a few months, but individual responses can vary.
Can I take Galafold while pregnant?
Consult your healthcare provider for guidance; it is generally advised to avoid use during pregnancy unless absolutely necessary.
What should I do if I miss a dose?
If you miss a dose, take it as soon as you remember. If it’s close to the time for your next dose, skip the missed dose and resume your regular schedule.
Are there any specific storage instructions for Galafold?
Store at room temperature, away from moisture and direct sunlight. Keep out of reach of children.
Is Galafold safe for long-term use?
Galafold is generally safe for long-term use, but regular monitoring and follow-up with a healthcare provider are important to ensure ongoing effectiveness and safety.
Comprehensive Drug Guide
How Does Galafold Work?
Galafold acts as a substrate reduction therapy by inhibiting the enzyme glucosylceramide synthase, leading to decreased synthesis of Gb3. This action helps to lower the accumulation of Gb3 in the body’s tissues, thereby reducing the symptoms and progression of Fabry disease.
What is Galafold Used For?
Treatment of adult patients with Fabry disease who have an amenable mutation.
Common Dosages
Galafold is available in the following form:
- Capsules: Typically available in a strength of 123 mg.
Typical Dosing
Who Shouldn't Take Galafold?
- Hypersensitivity: Known hypersensitivity to miglustat or any component of the formulation.
- Pregnancy: Women who are pregnant or planning to become pregnant should consult their healthcare provider.
Advice From The Pharmacist
Hydration: Maintain adequate hydration, as Fabry disease can affect kidney function.
Side Effects of Galafold
Common Side Effects
Diarrhea
Nausea
Abdominal pain
Headache
Uncommon/Severe Side Effects
Hypersensitivity reactions: Such as rash or difficulty breathing.
Kidney function changes: Monitor for signs of kidney impairment.
Alternatives to Galafold
Enzyme Replacement Therapy (ERT): Such as agalsidase beta or agalsidase alfa, which provide the missing enzyme directly.
