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Showing 1 to 10 of 12
Title Reference number Published Last updated
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 Reference number:HST12 HST00012 Published:27 November 2019 20191127 Last updated:27 November 2019 20191127
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations Reference number:HST11 HST00011 Published:09 October 2019 20191009 Last updated:09 October 2019 20191009
Patisiran for treating hereditary transthyretin amyloidosis Reference number:HST10 HST00010 Published:14 August 2019 20190814 Last updated:14 August 2019 20190814
Inotersen for treating hereditary transthyretin amyloidosis Reference number:HST9 HST00009 Published:22 May 2019 20190522 Last updated:22 May 2019 20190522
Burosumab for treating X-linked hypophosphataemia in children and young people Reference number:HST8 HST00008 Published:10 October 2018 20181010 Last updated:10 October 2018 20181010
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency Reference number:HST7 HST00007 Published:07 February 2018 20180207 Last updated:07 February 2018 20180207
Asfotase alfa for treating paediatric-onset hypophosphatasia Reference number:HST6 HST00006 Published:02 August 2017 20170802 Last updated:02 August 2017 20170802
Eliglustat for treating type 1 Gaucher disease Reference number:HST5 HST00005 Published:28 June 2017 20170628 Last updated:28 June 2017 20170628
Migalastat for treating Fabry disease Reference number:HST4 HST00004 Published:22 February 2017 20170222 Last updated:22 February 2017 20170222
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene Reference number:HST3 HST00003 Published:20 July 2016 20160720 Last updated:20 July 2016 20160720