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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. 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

  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. 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. 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

  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

  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. 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

  9. Docetaxel for the treatment of hormone-refractory metastatic prostate cancer (TA101)

    Evidence-based recommendations on docetaxel (Taxotere) for the treatment of hormone-refractory metastatic (advanced) prostate cancer

  10. Peginterferon alfa and ribavirin for the treatment of mild chronic hepatitis C (TA106)

    Evidence-based recommendations on peginterferon alfa (Pegasys) and ribavirin (Copegus) for treating mild chronic hepatitis C

  11. Capecitabine and oxaliplatin in the adjuvant treatment of stage III (Dukes' C) colon cancer (TA100)

    Evidence-based recommendations on capecitabine and oxaliplatin in the adjuvant treatment of stage III (Dukes' C) colon cancer in adults

  12. Ranibizumab and pegaptanib for the treatment of age-related macular degeneration (TA155)

    Evidence-based recommendations on ranibizumab (Lucentis) and pegaptanib (Macugen) for the treatment of age-related macular degeneration (AMD)

  13. Routine antenatal anti-D prophylaxis for women who are rhesus D negative (TA156)

    Evidence-based recommendations on routine antenatal anti-D prophylaxis for women who are rhesus D negative

  14. Dabigatran etexilate for the prevention of venous thromboembolism after hip or knee replacement surgery in adults (TA157)

    Evidence-based recommendations on dabigatran etexilate (Pradaxa) for the prevention of venous thromboembolism after hip or knee replacement surgery