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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. 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

  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. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

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

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia 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. 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. Pembrolizumab with pemetrexed and platinum chemotherapy for untreated, metastatic, non-squamous non-small-cell lung cancer (TA683)

    Evidence-based recommendations on pembrolizumab (Keytruda) with pemetrexed and platinum chemotherapy for untreated, metastatic, non-squamous non-small-cell lung

  10. Adalimumab, etanercept, infliximab, certolizumab pegol, golimumab, tocilizumab and abatacept for rheumatoid arthritis not previously treated with DMARDs or after conventional DMARDs only have failed (TA375)

    Evidence-based recommendations on adalimumab (Humira), etanercept (Enbrel), infliximab (Remicade, Remsima, Inflectra), certolizumab pegol (Cimzia), golimumab…

  11. Ciclosporin for treating dry eye disease that has not improved despite treatment with artificial tears (TA369)

    Evidence-based recommendations on ciclosporin (Ikervis) for treating dry eye disease that has not improved despite treatment with artificial tears

  12. Bortezomib for previously untreated mantle cell lymphoma (TA370)

    Evidence-based recommendations on bortezomib (Velcade) for treating previously untreated mantle cell lymphoma in adults

  13. Abatacept, adalimumab, etanercept and tocilizumab for treating juvenile idiopathic arthritis (TA373)

    Evidence-based recommendations on abatacept, adalimumab, etanercept and tocilizumab for treating polyarticular juvenile idiopathic arthritis (JIA)

  14. Dexamethasone intravitreal implant for treating diabetic macular oedema (TA349)

    Evidence-based recommendations on dexamethasone intravitreal implant (Ozurdex) for treating diabetic macular oedema

  15. Secukinumab for treating moderate to severe plaque psoriasis (TA350)

    Evidence-based recommendations on secukinumab (Cosentyx) for treating moderate to severe plaque psoriasis

  16. Vedolizumab for treating moderately to severely active Crohn's disease after prior therapy (TA352)

    Evidence-based recommendations on vedolizumab (Entyvio) for moderately to severely active Crohn's disease if TNF-alpha therapy has not worked or...

  17. Edoxaban for treating and for preventing deep vein thrombosis and pulmonary embolism (TA354)

    Evidence-based recommendations on edoxaban (Lixiana) for treating and preventing deep vein thrombosis (DVT) and pulmonary embolism

  18. Obinutuzumab in combination with chlorambucil for untreated chronic lymphocytic leukaemia (TA343)

    Evidence-based recommendations on obinutuzumab (Gazyvaro) with chlorambucil for treating untreated chronic lymphocytic leukaemia

  19. Everolimus for preventing organ rejection in liver transplantation (TA348)

    Evidence-based recommendations on everolimus (Certican) for preventing organ rejection in people having a liver transplant

  20. Edoxaban for preventing stroke and systemic embolism in people with non-valvular atrial fibrillation (TA355)

    Evidence-based recommendations on edoxaban (Lixiana) for preventing stroke and systemic embolism in people with non-valvular atrial fibrillation (AF)