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  1. Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)

    Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older

  2. 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

  3. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)

    Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

  4. Givosiran for treating acute hepatic porphyria (HST16)

    Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over

  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. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

  7. 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

  8. 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

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

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

  10. 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

  11. 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

  12. 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

  13. Guidance on the use of electroconvulsive therapy (TA59)

    Evidence-based recommendations on electroconvulsive therapy (ECT) for treating catatonia, prolonged or severe manic episodes or schizophrenia in adults

  14. Fulvestrant for untreated locally advanced or metastatic oestrogen-receptor positive breast cancer (TA503)

    Evidence-based recommendations on fulvestrant (Faslodex) for untreated locally advanced or metastatic (secondary) oestrogen-receptor positive breast cancer in

  15. Ceritinib for untreated ALK-positive non-small-cell lung cancer (TA500)

    Evidence-based recommendations on ceritinib (Zykadia) for untreated anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer (NSCLC)

  16. Holoclar for treating limbal stem cell deficiency after eye burns (TA467)

    Evidence-based recommendations on Holoclar for treating limbal stem cell deficiency in adults after eye burns

  17. Guidance on the use of glycoprotein IIb/IIIa inhibitors in the treatment of acute coronary syndromes (TA47)

    Evidence-based recommendations on glycoprotein IIb/IIIa inhibitors(abciximab [ReoPro], eptifibatide [Integrilin], tirofiban [Aggrastat]) for acute coronary

  18. Inhaled corticosteroids for the treatment of chronic asthma in children under the age of 12 years (TA131)

    Evidence-based recommendations on inhaled corticosteroids (ICSs) for chronic asthma in children aged under 12

  19. Darvadstrocel for treating complex perianal fistulas in Crohn's disease (TA556)

    Evidence-based recommendations on darvadstrocel (Alofisel; stem-cell treatment) for complex perianal fistulas with mildly or non-active luminal Crohn’s disease

  20. Autologous chondrocyte implantation using chondrosphere for treating symptomatic articular cartilage defects of the knee (TA508)

    Evidence-based recommendations on autologous chondrocyte implantation (ACI) using chondrosphere (Spherox) for symptomatic articular knee cartilage defects

  21. Regorafenib for previously treated unresectable or metastatic gastrointestinal stromal tumours (TA488)

    Evidence-based recommendations on on regorafenib (Stivarga) for previously treated gastrointestinal stromal tumours in adults

  22. Autologous chondrocyte implantation for treating symptomatic articular cartilage defects of the knee (TA477)

    Evidence-based recommendations on autologous chondrocyte implantation (ACI) in people with symptomatic articular cartilage defects of the knee

  23. Fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis (TA756)

    Evidence-based recommendations on fedratinib (Inrebic) for treating disease-related splenomegaly or symptoms in myelofibrosis in adults

  24. Brentuximab vedotin for treating CD30-positive Hodgkin lymphoma (TA524)

    Evidence-based recommendations on brentuximab vedotin (Adcetris) for treating CD30-positive Hodgkin lymphoma in adults

  25. Eribulin for treating locally advanced or metastatic breast cancer after 1 chemotherapy regimen (TA515)

    Evidence-based recommendations on eribulin (Halaven) for treating locally advanced or metastatic (secondary) breast cancer in adults who have had only 1 course

  26. 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