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Showing 1 to 6 of 6
Title Reference number Published Last updated
Asfotase alfa for treating paediatric-onset hypophosphatasia Reference number:HST6 HST00006 Published:August 2017 20170801 Last updated:August 2017 20170802
Eliglustat for treating type 1 Gaucher disease Reference number:HST5 HST00005 Published:June 2017 20170601 Last updated:June 2017 20170628
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