Search results

  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. 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

  3. 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

  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. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

    Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children

  7. 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

  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. HeartFlow FFRCT for estimating fractional flow reserve from coronary CT angiography (MTG32)

    Evidence-based recommendations on HeartFlow FFRCT for estimating fractional flow reserve from coronary CT angiography

  10. Virtual Touch Quantification to diagnose and monitor liver fibrosis in chronic hepatitis B and C (MTG27)

    Evidence-based recommendations on Virtual Touch Quantification to diagnose and monitor liver fibrosis in chronic hepatitis B and C

  11. Spectra Optia for automatic red blood cell exchange in people with sickle cell disease (MTG28)

    Evidence-based recommendations on Spectra Optia for automated red blood cell exchange in people with sickle cell disease

  12. XprESS multi sinus dilation system for treating chronic sinusitis (MTG30)

    Evidence-based recommendations on the XprESS multi-sinus dilation system for treating chronic sinusitis after medical treatment has failed

  13. Senza spinal cord stimulation system for delivering HF10 therapy to treat chronic neuropathic pain (MTG41)

    Evidence-based recommendations on the Senza spinal cord stimulation system for delivering HF10 therapy to treat chronic neuropathic pain

  14. MiraQ for assessing graft flow during coronary artery bypass graft surgery (MTG8)

    Evidence-based recommendations on MiraQ for assessing graft flow during coronary artery bypass graft surgery

  15. PleurX peritoneal catheter drainage system for vacuum-assisted drainage of treatment-resistant, recurrent malignant ascites (MTG9)

    Evidence-based recommendations on PleurX for vacuum-assisted drainage of treatment-resistant, recurrent malignant ascites