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Showing 1 to 7 of 7
| Title | Reference number | Reference number sort | Published | Published date sort | Last updated | Last updated sort |
|---|---|---|---|---|---|---|
| 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 |
| Eculizumab for treating atypical haemolytic uraemic syndrome | Reference number:HST1 | HST00001 | Published:January 2015 | 20150101 | Last updated:January 2015 | 20150128 |