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  1. 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

  2. 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

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

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

  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. Avelumab with axitinib for untreated advanced renal cell carcinoma (TA645)

    Evidence-based recommendations on avelumab (Bavencio) with axitinib (Inlyta) for untreated advanced renal cell carcinoma in adults

  10. Avatrombopag for treating thrombocytopenia in people with chronic liver disease needing a planned invasive procedure (TA626)

    Evidence-based recommendations on avatrombopag (Doptelet) for treating severe thrombocytopenia in adults with chronic liver disease needing a planned invasive

  11. Treosulfan with fludarabine for malignant disease before allogeneic stem cell transplant (TA640)

    Evidence-based recommendations on treosulfan (Trecondi) with fludarabine for conditioning treatment before allogeneic haematopoietic stem cell transplant for

  12. Entrectinib for treating ROS1-positive advanced non-small-cell lung cancer (TA643)

    Evidence-based recommendations on entrectinib (Rozlytrek) for ROS1-positive advanced non-small-cell lung cancer (NSCLC) in adults who have not had ROS1

  13. Entrectinib for treating NTRK fusion-positive solid tumours (TA644)

    Evidence-based recommendations on entrectinib (Rozlytrek) for treating neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours in adults and

  14. Lutetium (177Lu) oxodotreotide for treating unresectable or metastatic neuroendocrine tumours (TA539)

    Evidence-based recommendations on lutetium (177Lu) oxodotreotide (Lutathera) for treating unresectable or metastatic neuroendocrine tumours in adults

  15. Brentuximab vedotin in combination for untreated systemic anaplastic large cell lymphoma (TA641)

    Evidence-based recommendations on brentuximab vedotin (Adcetris) with cyclophosphamide, doxorubicin and prednisone for untreated systemic anaplastic large cell

  16. Gilteritinib for treating relapsed or refractory acute myeloid leukaemia (TA642)

    Evidence-based recommendations on gilteritinib (Xospata) for relapsed or refractory FLT3-mutation-positive acute myeloid leukaemia in adults

  17. Guidance on the use of drugs for early thrombolysis in the treatment of acute myocardial infarction (TA52)

    Evidence-based recommendations on streptokinase (Streptase), alteplase (Actilyse), reteplase (Rapilysin) and tenecteplase (Metalyse) for early thrombolysis in

  18. Pemetrexed for the maintenance treatment of non-small-cell lung cancer (TA190)

    Evidence-based recommendations on pemetrexed disodium (Alimta) for the maintenance treatment of non-small-cell lung cancer