Published Guidance, NICE advice and quality standards
Showing 21 to 27 of 27
| Title | Reference number | Published | Last updated |
|---|---|---|---|
| Patisiran for treating hereditary transthyretin amyloidosis | HST10 | ||
| Inotersen for treating hereditary transthyretin amyloidosis | HST9 | ||
| Burosumab for treating X-linked hypophosphataemia in children and young people | HST8 | ||
| Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency | HST7 | ||
| Eliglustat for treating type 1 Gaucher disease | HST5 | ||
| Migalastat for treating Fabry disease | HST4 | ||
| Eculizumab for treating atypical haemolytic uraemic syndrome | HST1 |