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  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  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. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

  8. 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

  9. Carmustine implants and temozolomide for the treatment of newly diagnosed high-grade glioma (TA121)

    Evidence-based recommendations on carmustine implants Gliadel) and temozolomide (Temodal) for newly diagnosed high-grade glioma (brain tumour)

  10. Pembrolizumab for treating advanced melanoma after disease progression with ipilimumab (TA357)

    Evidence-based recommendations on pembrolizumab (Keytruda) for treating advanced melanoma after disease progression with ipilimumab in adults

  11. Tolvaptan for treating autosomal dominant polycystic kidney disease (TA358)

    Evidence-based recommendations on tolvaptan (Jinarc) for treating autosomal dominant polycystic kidney disease (ADPKD)

  12. Idelalisib for treating chronic lymphocytic leukaemia (TA359)

    Evidence-based recommendations on idelalisib (Zydelig) for treating chronic lymphocytic leukaemia (CLL) in adults

  13. Ledipasvir–sofosbuvir for treating chronic hepatitis C (TA363)

    Evidence-based recommendations on ledipasvir–sofosbuvir (Harvoni) for treating some types (genotypes) of chronic hepatitis C

  14. 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...

  15. 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

  16. 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)

  17. Atezolizumab for treating locally advanced or metastatic non-small-cell lung cancer after chemotherapy (TA520)

    Evidence-based recommendations on atezolizumab (Tecentriq) for locally advanced or metastatic non-small-cell lung cancer after chemotherapy in adults

  18. Pembrolizumab for treating locally advanced or metastatic urothelial carcinoma after platinum-containing chemotherapy (TA519)

    Evidence-based recommendations on pembrolizumab (Keytruda) for previously treated locally advanced or metastatic urothelial carcinoma in adults

  19. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma (TA505)

    Evidence-based recommendations on ixazomib (Ninlaro), with lenalidomide and dexamethasone for relapsed or refractory multiple myeloma in adults

  20. Atezolizumab for untreated PD-L1-positive locally advanced or metastatic urothelial cancer when cisplatin is unsuitable (TA492)

    Evidence-based recommendations on atezolizumab (Tecentriq) for untreated PD-L1-positive locally advanced or metastatic urothelial carcinoma in adults