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Showing 781 to 795 of 7675 results
Patisiran for treating hereditary transthyretin amyloidosis (HST10)
Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy.
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children.
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.