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Volanesorsen for treating familial chylomicronaemia syndrome (HST13)
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults.
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults.
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.
Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children.
Setmelanotide for treating obesity caused by LEPR or POMC deficiency (HST21)
Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC deficiency in people 6 years and over.
Evidence-based recommendations on ataluren (Translarna) for Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in babies, children, young people and adults.
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating presymptomatic spinal muscular atrophy in babies aged 12 months and under.
Evidence-based recommendations on lumasiran (Oxlumo) for primary hyperoxaluria type 1 in people of all ages.
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over.
Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older.
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children.
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages.
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over.
Afamelanotide for treating erythropoietic protoporphyria (HST27)
Evidence-based recommendations on afamelanotide (Scenesse) for treating erythropoietic protoporphyria in adults.