Showing 1 to 10 of 12
| Title | Reference number | Reference number sort | Published | Published date sort | Last updated | Last updated sort |
|---|---|---|---|---|---|---|
| Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 | Reference number:HST12 | HST00012 | Published:November 2019 | 20191101 | Last updated:November 2019 | 20191127 |
| 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 |