Migalastat: A Review in Fabry Disease

Author:

McCafferty Emma H.,Scott Lesley J.

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical)

Reference61 articles.

1. Ortiz A, Germain DP, Desnick RJ, et al. Fabry disease revisited: management and treatment recommendations for adult patients. Mol Genet Metab. 2018;123(4):416–27.

2. Amicus Therapeutics. Galafold™ (migalastat) capsules, for oral use: US prescribing information. 2018. https://www.fda.gov/ . Accessed 18 Oct 2018.

3. European Medicines Agency. Migalastat (Galafold): EU summary of product characteristics. 2018. https://www.ema.europa.eu/ . Accessed 18 Oct 2018.

4. Brady RO, Gal AE, Bradley RM, et al. Enzymatic defect in Fabry’s disease. Ceramidetrihexosidase deficiency. N Engl J Med. 1967;276(21):1163–7.

5. National Institute for Health and Care Excellence. Migalastat for treating Fabry disease. 2017. https://www.nice.org.uk/guidance/hst4 . Accessed 26 Oct 2018.

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