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
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Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
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Givosiran for treating acute hepatic porphyria (HST16)
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over
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Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
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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
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Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children