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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. 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. 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

  4. 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

  5. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

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

  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. 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

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

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

  9. 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

  10. 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

  11. 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

  12. 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

  13. Tucatinib with trastuzumab and capecitabine for treating HER2-positive advanced breast cancer after 2 or more anti-HER2 therapies (TA786)

    Evidence-based recommendations on tucatinib (TUKYSA) for HER2 positive locally advanced or metastatic breast cancer in adults after 2 or more anti HER2 treatment therapies

  14. Venetoclax with low dose cytarabine for untreated acute myeloid leukaemia when intensive chemotherapy is unsuitable (TA787)

    Evidence-based recommendations on venetoclax (Venclyxto) with low dose cytarabine for untreated acute myeloid leukaemia in adults when intensive chemotherapy is unsuitable

  15. Cochlear implants for children and adults with severe to profound deafness (TA566)

    Evidence-based recommendations on cochlear implants for children and adults with severe to profound deafness

  16. Benralizumab for treating severe eosinophilic asthma (TA565)

    Evidence-based recommendations on benralizumab (Fasenra) for treating severe eosinophilic asthma (SEA; elevated levels of eosinophils in blood) in adults

  17. Regorafenib for previously treated advanced hepatocellular carcinoma (TA555)

    Evidence-based recommendations on regorafenib (Stivarga) for treating advanced hepatocellular carcinoma (liver cancer) in adults who have had sorafenib