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  1. 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

  2. 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

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

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

  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. 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

  6. 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

  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. 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

  10. 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

  11. 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

  12. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

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

  13. 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

  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. 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

  16. Apixaban for preventing stroke and systemic embolism in people with non-valvular atrial fibrillation (TA275)

    Evidence-based recommendations on apixaban (Eliquis) for preventing stroke and systemic embolism in adults with non-valvular atrial fibrillation

  17. Talimogene laherparepvec for treating unresectable metastatic melanoma (TA410)

    Evidence-based recommendations on talimogene laherparepvec (Imlygic) for treating unresectable metastatic melanoma in adults when systemically administered

  18. Sotagliflozin with insulin for treating type 1 diabetes (TA622)

    Evidence-based recommendations on sotagliflozin with insulin for treating type 1 diabetes in adults with a body mass index (BMI) of at least 27 kg/m2

  19. Palbociclib with fulvestrant for treating hormone receptor-positive, HER2-negative, advanced breast cancer (TA619)

    Evidence-based recommendations on palbociclib (Ibrance) with fulvestrant for hormone receptor-positive, HER2-negative locally advanced/metastatic breast cancer

  20. Ustekinumab for moderately to severely active Crohn's disease after previous treatment (TA456)

    Evidence-based recommendations on ustekinumab (Stelara) for previously treated moderately to severely active Crohn’s disease in adults

  21. 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

  22. 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

  23. 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

  24. 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

  25. 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

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

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