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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. 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. 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

  5. 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

  6. 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

  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. 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. 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

  12. Sofosbuvir–velpatasvir–voxilaprevir for treating chronic hepatitis C (TA507)

    Evidence-based recommendations on sofosbuvir–velpatasvir–voxilaprevir (Vosevi) for treating chronic hepatitis C (HCV) in adults

  13. 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

  14. Frequency of application of topical corticosteroids for atopic eczema (TA81)

    Evidence-based recommendations on the frequency of application of topical corticosteroids for treating atopic eczema (atopic dermatitis)

  15. Tacrolimus and pimecrolimus for atopic eczema (TA82)

    Evidence-based recommendations on non-steroidal tacrolimus and pimecrolimus for treating atopic eczema (atopic dermatitis)