Search results

  1. 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

  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. 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

  4. 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

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

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

  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. 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

  8. 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

  9. PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA Xpress PlGF 1-2-3 test, and BRAHMS sFlt-1 Kryptor/BRAHMS PlGF plus Kryptor PE ratio) (DG23)

    Evidence-based recommendations on PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA

  10. Integrated multiplex PCR tests for identifying gastrointestinal pathogens in people with suspected gastroenteritis (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B assay) (DG26)

    Evidence-based recommendations on integrated multiplex polymerase chain tests (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B