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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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  3. 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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  6. 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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  8. 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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  13. 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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  14. 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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  16. 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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  18. 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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  19. 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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  20. 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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  38. Procalcitonin testing for diagnosing and monitoring sepsis (ADVIA Centaur BRAHMS PCT assay, BRAHMS PCT Sensitive Kryptor assay, Elecsys BRAHMS PCT assay, LIAISON BRAHMS PCT assay and VIDAS BRAHMS PCT assay) (DG18)

    Evidence-based recommendations on procalcitonin testing for diagnosing and monitoring sepsis (ADVIA Centaur BRAHMS PCT assay, BRAHMS PCT Sensitive

    Diagnostics guidance
    Published

  40. SepsiTest assay for rapidly identifying bloodstream bacteria and fungi (DG20)

    Evidence-based recommendations on the SepsiTest assay for rapidly identifying bloodstream bacteria and fungi

    Diagnostics guidance
    Last updated

  41. Integrated sensor-augmented pump therapy systems for managing blood glucose levels in type 1 diabetes (the MiniMed Paradigm Veo system and the Vibe and G4 PLATINUM CGM system) (DG21)

    Evidence-based recommendations on the MiniMed Paradigm Veo and Vibe and G4 PLATINUM CGM sensor-augmented pump therapy systems, which combine continuous glucose

    Diagnostics guidance
    Published
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  44. PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA Xpress PlGF 1-2-3 test, and BRAHMS sFlt-1 Kryptor/BRAHMS PlGF plus Kryptor PE ratio) (DG23)

    Evidence-based recommendations on PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA

    Diagnostics guidance
    Published
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  46. EarlyCDT Lung for assessing risk of lung cancer in solid lung nodules (DG46)

    Evidence-based recommendations on EarlyCDT Lung for assessing the risk of lung cancer in solid lung nodules

    Diagnostics guidance
    Published
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  47. PredictSURE IBD and IBDX to guide treatment of Crohn's disease (DG45)

    Evidence-based recommendations on PredictSURE IBD and IBDX for guiding treatment of Crohn’s disease

    Diagnostics guidance
    Published
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  49. Integrated multiplex PCR tests for identifying gastrointestinal pathogens in people with suspected gastroenteritis (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B assay) (DG26)

    Evidence-based recommendations on integrated multiplex polymerase chain tests (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B

    Diagnostics guidance
    Published
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  50. Quantitative faecal immunochemical tests to guide referral for colorectal cancer in primary care (DG30)

    Evidence-based recommendations on faecal immunochemical tests (OC Sensor, HM-JACKarc, FOB Gold and RIDASCREEN) to guide GP referral for colorectal cancer

    Diagnostics guidance
    Published
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