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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8 results
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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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Metreleptin for treating lipodystrophy (HST14)
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
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Volanesorsen for treating familial chylomicronaemia syndrome (HST13)
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
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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
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Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherit
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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
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Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis