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Showing 1 to 10 of 11
Title Reference number Published Last updated
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations Reference number:HST11 HST00011 Published:October 2019 20191001 Last updated:October 2019 20191009
Patisiran for treating hereditary transthyretin amyloidosis Reference number:HST10 HST00010 Published:August 2019 20190801 Last updated:August 2019 20190814
Inotersen for treating hereditary transthyretin amyloidosis Reference number:HST9 HST00009 Published:May 2019 20190501 Last updated:May 2019 20190522
Burosumab for treating X-linked hypophosphataemia in children and young people Reference number:HST8 HST00008 Published:October 2018 20181001 Last updated:October 2018 20181010
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency Reference number:HST7 HST00007 Published:February 2018 20180201 Last updated:February 2018 20180207
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