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Long\u2010term treatment may slow down disease progression, but cardiac, renal, and cerebral complications still develop in most patients. In addition, lifelong intravenous treatment is burdensome. Therefore, several new treatment approaches have been explored over the past decade. Chaperone therapy (Migalastat; 1\u2010deoxygalactonojirimycin) is the only other currently approved therapy for Fabry disease. This oral small molecule aims to improve enzyme activity of mutated \u03b1\u2010galactosidase A and can only be used in patients with specific mutations. Treatments currently under evaluation in (pre)clinical trials are second generation enzyme replacement therapies (Pegunigalsidase\u2010alfa, Moss\u2010aGal), substrate reduction therapies (Venglustat and Lucerastat), mRNA\u2010 and gene\u2010based therapy. This review summarises the knowledge on currently available and potential future options for the treatment of Fabry disease.<\/jats:p>","DOI":"10.1002\/jimd.12228","type":"journal-article","created":{"date-parts":[[2020,2,21]],"date-time":"2020-02-21T06:52:52Z","timestamp":1582267972000},"page":"908-921","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":141,"title":["Developments in the treatment of Fabry disease"],"prefix":"10.1002","volume":"43","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4745-5718","authenticated-orcid":false,"given":"Sanne J.","family":"van der Veen","sequence":"first","affiliation":[{"name":"Department of Endocrinology and Metabolism Amsterdam UMC, University of Amsterdam AZ Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla E. 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