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. Alpha Stim AID (MT477)

    In development [GID-MT542] Expected publication date: 08 March 2021

  10. Point-of-care creatinine devices to assess kidney function before CT imaging with intravenous contrast (DG37)

    Evidence-based recommendations on point-of-care creatinine devices to assess kidney function before CT imaging with intravenous contrast. The tests are

  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

  13. Tumour profiling tests to guide adjuvant chemotherapy decisions in early breast cancer (DG34)

    Evidence-based recommendations on tumour profiling tests to guide adjuvant chemotherapy decisions for people with early breast cancer. The tests are EndoPredict

  14. SonoVue (sulphur hexafluoride microbubbles) – contrast agent for contrast-enhanced ultrasound imaging of the liver (DG5)

    Evidence-based recommendations on SonoVue (sulphur hexafluoride microbubbles) for contrast-enhanced ultrasound imaging of the liver