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  2. Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

    Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people aged

    Highly specialised technologies guidance
    Published
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  5. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

    Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

    Highly specialised technologies guidance
    Published
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  7. 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

    Highly specialised technologies guidance
    Published
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  9. 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

    Highly specialised technologies guidance
    Published
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  10. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

    Highly specialised technologies guidance
    Published
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  11. 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

    Highly specialised technologies guidance
    Published
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  12. Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

    Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

    Highly specialised technologies guidance
    Published
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  14. Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

    Highly specialised technologies guidance
    Published
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  16. 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

    Highly specialised technologies guidance
    Published
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  17. 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

    Highly specialised technologies guidance
    Published
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  18. 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

    Highly specialised technologies guidance
    Published
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  20. 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

    Highly specialised technologies guidance
    Published
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  41. HeartFlow FFRCT for estimating fractional flow reserve from coronary CT angiography (MTG32)

    Evidence-based recommendations on HeartFlow FFRCT for estimating fractional flow reserve from coronary CT angiography

    Medical technologies guidance
    Last updated
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  44. DyeVert Systems for reducing the risk of acute kidney injury in coronary and peripheral angiography (MTG60)

    Evidence-based recommendations on DyeVert Systems for reducing the risk of acute kidney injury in coronary and peripheral angiography

    Medical technologies guidance
    Published
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  46. ClearGuard HD antimicrobial barrier caps for preventing haemodialysis catheter-related bloodstream infections (MTG62)

    Evidence-based recommendations on ClearGuard HD antimicrobial barrier caps for preventing haemodialysis catheter-related bloodstream infections

    Medical technologies guidance
    Published
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