Published Guidance, NICE advice and quality standards
Showing 11 to 20 of 27
| Title | Reference number | Published | Last updated |
|---|---|---|---|
| Elosulfase alfa for treating mucopolysaccharidosis type 4A | HST19 | ||
| Givosiran for treating acute hepatic porphyria | HST16 | ||
| Inotersen for treating hereditary transthyretin amyloidosis | HST9 | ||
| Lumasiran for treating primary hyperoxaluria type 1 | HST25 | ||
| Metreleptin for treating lipodystrophy | HST14 | ||
| Migalastat for treating Fabry disease | HST4 | ||
| Odevixibat for treating progressive familial intrahepatic cholestasis | HST17 | ||
| Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy | HST24 | ||
| Onasemnogene abeparvovec for treating spinal muscular atrophy | HST15 | ||
| Patisiran for treating hereditary transthyretin amyloidosis | HST10 |