Pharmacological chaperone (α-galactosidase A)
Migalastat
Brand names: Galafold
Adult dose
Dose: 123mg PO every other day on empty stomach
Route: PO
Frequency: alternate days
Clinical pearls
- Fabry disease in patients with amenable mutation
- Specialist metabolic / renal — alternative to enzyme replacement
Contraindications
- Non-amenable GLA mutations
- eGFR <30
- Hypersensitivity
Side effects
- Headache
- UTI
- GI upset
- Proteinuria
Monitoring
- Renal function
- Cardiac function
- Disease biomarkers (Lyso-GL3)
Reference: BNF; NICE TA517; SmPC; https://bnf.nice.org.uk/drugs/migalastat/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Pathways
- Diabetic Ketoacidosis (DKA) · JBDS 2013 / Joint British Diabetes Societies; NICE NG17
- Adult Hypoglycaemia (Treated Diabetes) · JBDS-IP (2023): Hospital Management of Hypoglycaemia
- Adrenal Crisis · Society for Endocrinology Emergency Guidance (2024)
- Type 2 Diabetes Management · NICE NG28 2022
- Hyperthyroidism Management · BTA / ETA 2018
- Adrenal Insufficiency · Society of Endocrinology / ESE 2016