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  1. 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

  2. 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

  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. 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

  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. Alpha Stim AID

    In development [GID-MT542] Expected publication date: 27 January 2021

  10. SeHCAT (tauroselcholic [75 selenium] acid) for the investigation of diarrhoea due to bile acid malabsorption in people with diarrhoea-predominant irritable bowel syndrome (IBS-D) or Crohn's disease without ileal resection (DG7)

    Evidence-based recommendations on SeHCAT (tauroselcholic [75selenium] acid) for diagnosing bile acid malabsorption in people with IBS-D or Crohn’s disease

  11. Adjunctive colposcopy technologies for assessing suspected cervical abnormalities: the DYSIS colposcope with DYSISmap and the ZedScan I (DG32)

    Evidence-based recommendations on 2 adjunctive colposcopy technologies (the DYSIS colposcope with DYSISmap and the ZedScan I) for assessing suspected cervical

  12. Biomarker tests to help diagnose preterm labour in women with intact membranes (DG33)

    Evidence-based recommendations on biomarker tests to help diagnose preterm labour in women with intact membranes. The tests are Actim Partus, PartoSure and the