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  1. 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

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

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

  3. 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

  4. 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

  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. 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

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

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

  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. Trifluridine–tipiracil for treating metastatic gastric cancer or gastro-oesophageal junction adenocarcinoma after 2 or more therapies (TA669)

    Evidence-based recommendations on trifluridine–tipiracil (Lonsurf) for treating metastatic gastric cancer or gastro-oesophageal junction adenocarcinoma in adult

  10. Brigatinib for ALK-positive advanced non-small-cell lung cancer that has not been previously treated with an ALK inhibitor (TA670)

    Evidence-based recommendations on brigatinib (Alunbrig) for anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer that has not been

  11. Niraparib for maintenance treatment of advanced ovarian, fallopian tube and peritoneal cancer after response to first-line platinum-based chemotherapy (TA673)

    Evidence-based recommendations on niraparib for maintenance treatment of advanced (FIGO stages 3 and 4) high-grade epithelial ovarian, fallopian tube or primary

  12. Autologous anti-CD19-transduced CD3+ cells for treating relapsed or refractory mantle cell lymphoma (TA677)

    Evidence-based recommendations on autologous anti-CD19-transduced CD3+ cells for treating relapsed or refractory mantle cell lymphoma in adults who have

  13. Dapagliflozin for treating chronic heart failure with reduced ejection fraction (TA679)

    Evidence-based recommendations on dapagliflozin (Forxiga) for symptomatic chronic heart failure with reduced ejection fraction in adults