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  1. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

  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

  3. 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

  4. 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

  5. Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

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

  6. 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

  7. 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

  8. 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

  9. 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

  10. SonoVue (sulphur hexafluoride microbubbles) – contrast agent for contrast-enhanced ultrasound imaging of the liver (DG5)

    Evidence-based recommendations on SonoVue (sulphur hexafluoride microbubbles) for contrast-enhanced ultrasound imaging of the liver

  11. High-sensitivity troponin tests for the early rule out of NSTEMI (DG40)

    Evidence-based recommendations on high-sensitivity troponin tests for the early rule out of NSTEMI (non-ST-segment elevation myocardial infarction)

  12. Implantable cardiac monitors to detect atrial fibrillation after cryptogenic stroke (DG41)

    Evidence-based recommendations on implantable cardiac monitors to detect atrial fibrillation after cryptogenic stroke

  13. QAngio XA 3D QFR and CAAS vFFR imaging software for assessing coronary stenosis during invasive coronary angiography (DG43)

    Evidence-based recommendations on QAngio XA 3D QFR and CAAS vFFR imaging software for assessing coronary stenosis during invasive coronary angiography

  14. Tests to help assess risk of acute kidney injury for people being considered for critical care admission (ARCHITECT and Alinity i Urine NGAL assays, BioPorto NGAL test and NephroCheck test) (DG39)

    Evidence-based recommendations on tests to help assess risk of acute kidney injury for people being considered for critical care admission. The tests are the

  15. Faecal calprotectin diagnostic tests for inflammatory diseases of the bowel (DG11)

    Evidence-based recommendations on faecal calprotectin tests for distinguishing between inflammatory bowel diseases (such as Crohn’s disease and ulcerative

  16. Measuring fractional exhaled nitric oxide concentration in asthma: NIOX MINO, NIOX VERO and NObreath (DG12)

    Evidence-based recommendations on NIOX MINO, NIOX VERO and NObreath for measuring the amount of exhaled nitric oxide (FeNO) in the breath to help diagnose