Published: Guidance, quality standards and advice
Showing 26 to 30 of 30
| Title | Reference number | Published | Last updated |
|---|---|---|---|
| Setmelanotide for treating obesity caused by LEPR or POMC deficiency | HST21 | ||
| Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency | HST7 | ||
| Velmanase alfa for treating alpha-mannosidosis | HST29 | ||
| Volanesorsen for treating familial chylomicronaemia syndrome | HST13 | ||
| Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations | HST11 |