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Showing 1 to 4 of 4
Title Reference number Published Last updated
Migalastat for treating Fabry disease Reference number:HST4 HST00004 Published:February 2017 20170201 Last updated:February 2017 20170222
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene Reference number:HST3 HST00003 Published:July 2016 20160701 Last updated:July 2016 20160720
Elosulfase alfa for treating mucopolysaccharidosis type IVa Reference number:HST2 HST00002 Published:December 2015 20151201 Last updated:December 2015 20151216
Eculizumab for treating atypical haemolytic uraemic syndrome Reference number:HST1 HST00001 Published:January 2015 20150101 Last updated:January 2015 20150128