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  1. 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

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

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

  4. 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

  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. 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

  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. Apixaban for preventing stroke and systemic embolism in people with nonvalvular atrial fibrillation (TA275)

    Evidence-based recommendations on apixaban (Eliquis) for preventing stroke and systemic embolism in people with nonvalvular atrial fibrillation (AF)

  10. Colistimethate sodium and tobramycin dry powders for inhalation for treating pseudomonas lung infection in cystic fibrosis (TA276)

    Evidence-based recommendations on colistimethate sodium and tobramycin dry powders for inhalation for pseudomonas lung infection in cystic fibrosis

  11. Denosumab for the prevention of skeletal-related events in adults with bone metastases from solid tumours (TA265)

    Evidence-based recommendations on denosumab (XGEVA) for the prevention of skeletal-related events in adults with bone metastases from solid tumours

  12. Mannitol dry powder for inhalation for treating cystic fibrosis (TA266)

    Evidence-based recommendations on mannitol dry powder for inhalation (Bronchitol) for treating cystic fibrosis (CF)

  13. Ipilimumab for previously treated advanced (unresectable or metastatic) melanoma (TA268)

    Evidence-based recommendations on ipilimumab (Yervoy) for previously treated advanced (unresectable or metastatic) melanoma

  14. Percutaneous vertebroplasty and percutaneous balloon kyphoplasty for treating osteoporotic vertebral compression fractures (TA279)

    Evidence-based recommendations on percutaneous vertebroplasty and percutaneous balloon kyphoplasty for osteoporotic vertebral compression fractures

  15. Ranibizumab for treating visual impairment caused by macular oedema secondary to retinal vein occlusion (TA283)

    Evidence-based recommendations on ranibizumab (Lucentis) for treating sight problems caused by macular oedema secondary to retinal vein occlusion

  16. Bevacizumab in combination with paclitaxel and carboplatin for first-line treatment of advanced ovarian cancer (TA284)

    Evidence-based recommendations on bevacizumab (Avastin) in combination with paclitaxel and carboplatin for first-line treatment of advanced ovarian cancer

  17. Vinflunine for the treatment of advanced or metastatic transitional cell carcinoma of the urothelial tract (TA272)

    Evidence-based recommendations on vinflunine (Javlor) for the treatment of advanced or metastatic transitional cell carcinoma of the urothelial tract

  18. Bevacizumab in combination with gemcitabine and carboplatin for treating the first recurrence of platinum-sensitive advanced ovarian cancer (TA285)

    Evidence-based recommendations on bevacizumab (Avastin) in combination with gemcitabine and carboplatin for treating the first recurrence of

  19. Rivaroxaban for treating pulmonary embolism and preventing recurrent venous thromboembolism (TA287)

    Evidence-based recommendations on rivaroxaban (Xarelto) for treating pulmonary embolism and preventing recurrent venous thromboembolism