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

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

  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. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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