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  1. 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

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

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

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

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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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

  10. 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

  11. SeHCAT (tauroselcholic [75 selenium] acid) for the investigation of diarrhoea due to bile acid malabsorption in people with diarrhoea-predominant irritable bowel syndrome (IBS-D) or Crohn's disease without ileal resection (DG7)

    Evidence-based recommendations on SeHCAT (tauroselcholic [75selenium] acid) for diagnosing bile acid malabsorption in people with IBS-D or Crohn’s disease

  12. 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

  13. 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)

  14. 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