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Fabry disease: Difference between revisions
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Fabry disease is a genetic condition involving lysosomal storage (E75.2)<ref>https://de.wikipedia.org/wiki/Morbus_Fabry</ref> | Fabry disease is a genetic condition involving lysosomal storage (E75.2)<ref>https://de.wikipedia.org/wiki/Morbus_Fabry</ref>is an X-linked disorder caused by alpha-galactosidase A deficiency | ||
<ref>https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0877-4 | |||
</ref> | |||
<gallery>File:Morbus Fabry affected organs.svg.png</gallery> | <gallery>File:Morbus Fabry affected organs.svg.png</gallery> | ||
[[migalastat]] or 1-deoxygalactonojirimycin received an orphan drug approval | [[migalastat]] or 1-deoxygalactonojirimycin received an orphan drug approval | ||
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[https://www.ncbi.nlm.nih.gov/pubmed/30064518 Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY] | [https://www.ncbi.nlm.nih.gov/pubmed/30064518 Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY] | ||
<references/> | <references/> | ||
Revision as of 00:44, 11 August 2018
Fabry disease is a genetic condition involving lysosomal storage (E75.2)[1]is an X-linked disorder caused by alpha-galactosidase A deficiency [2]
migalastat or 1-deoxygalactonojirimycin received an orphan drug approval [3]