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

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

  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. 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

  4. 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

  5. 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

  6. 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

  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. 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. Brentuximab vedotin for treating relapsed or refractory systemic anaplastic large cell lymphoma (TA478)

    Evidence-based recommendations on brentuximab vedotin (Adcetris) for treating relapsed or refractory systemic anaplastic large cell lymphoma in adults

  10. 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

  11. Paclitaxel as albumin-bound nanoparticles with gemcitabine for untreated metastatic pancreatic cancer (TA476)

    Evidence-based recommendations on paclitaxel as albumin-bound nanoparticles (nab-paclitaxel; Abraxane) for untreated metastatic pancreatic cancer in adults

  12. Cetuximab for treating recurrent or metastatic squamous cell cancer of the head and neck (TA473)

    Evidence-based recommendations on cetuximab (Erbitux) for treating recurrent or metastatic squamous cell cancer of the head and neck in adults

  13. Palbociclib with an aromatase inhibitor for previously untreated, hormone receptor-positive, HER2-negative, locally advanced or metastatic breast cancer (TA495)

    Evidence-based recommendations on palbociclib (Ibrance) as initial endocrine-based therapy for hormone receptor-positive, HER2-negative, locally advanced or

  14. Ibrutinib for treating Waldenstrom's macroglobulinaemia (TA491)

    Evidence-based recommendations on ibrutinib (Imbruvica) for treating Waldenstrom's macroglobulinaemia (non-Hodgkin lymphoma) in adults after at least 1 therapy

  15. 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