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  1. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

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

  2. 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

  3. 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

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

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

  5. 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

  6. 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

  7. 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

  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

  9. 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

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

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

  11. 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

  12. Setmelanotide for treating obesity caused by LEPR or POMC deficiency (HST21)

    Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC deficiency in people 6 years and over

  13. 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

  14. Multiple frequency bioimpedance devices to guide fluid management in people with chronic kidney disease having dialysis (DG29)

    Evidence-based recommendations on multiple frequency bioimpedance devices to guide fluid management in people with chronic kidney disease having dialysis

  15. New generation cardiac CT scanners (Aquilion ONE, Brilliance iCT, Discovery CT750 HD and Somatom Definition Flash) for cardiac imaging in people with suspected or known coronary artery disease in whom imaging is difficult with earlier generation CT scanners (DG3)

    Evidence-based recommendations on new generation cardiac CT scanners (Aquilion ONE, Brilliance iCT, Discovery CT750 HD and Somatom Definition Flash) for cardiac

  16. Biomarker tests to help diagnose preterm labour in women with intact membranes (DG33)

    Evidence-based recommendations on biomarker tests to help diagnose preterm labour in women with intact membranes. The tests are Actim Partus, PartoSure and the

  17. 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

  18. Therapeutic monitoring of TNF-alpha inhibitors in rheumatoid arthritis (DG36)

    Evidence-based recommendations on enzyme-linked immunosorbent assay (ELISA) tests for therapeutic monitoring of tumour necrosis factor (TNF)-alpha inhibitors in

  19. Tumour profiling tests to guide adjuvant chemotherapy decisions in early breast cancer (DG34)

    Evidence-based recommendations on tumour profiling tests to guide adjuvant chemotherapy decisions for people with early breast cancer. The tests are EndoPredict

  20. SeHCAT (tauroselcholic [75 selenium] acid) for diagnosing bile acid diarrhoea (DG44)

    Evidence-based recommendations on SeHCAT (tauroselcholic [75 selenium] acid) for diagnosing bile acid diarrhoea

  21. Rapid tests for group A streptococcal infections in people with a sore throat (DG38)

    Evidence-based recommendations on rapid tests for group A streptococcal infections in people aged 5 and over with a sore throat

  22. Lead-I ECG devices for detecting symptomatic atrial fibrillation using single time point testing in primary care (DG35)

    Evidence-based recommendations on lead-I electrocardiogram (ECG) devices (imPulse, Kardia Mobile, MyDiagnostick and Zenicor-ECG) for detecting symptomatic

  23. Atrial fibrillation and heart valve disease: self-monitoring coagulation status using point-of-care coagulometers (the CoaguChek XS system) (DG14)

    Evidence-based recommendations on the CoaguChek XS system for self-monitoring coagulation status in adults and children. The recommendations originally included

  24. Molecular testing strategies for Lynch syndrome in people with colorectal cancer (DG27)

    Evidence-based recommendations on using immunohistochemistry or microsatellite instability testing to guide further testing for Lynch syndrome in people with