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  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. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)

    Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

  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. Givosiran for treating acute hepatic porphyria (HST16)

    Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over

  5. Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)

    Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older

  6. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

  7. 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

  8. 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

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

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

  10. 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

  11. 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

  12. 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

  13. ClearGuard HD antimicrobial barrier caps for preventing haemodialysis catheter-related bloodstream infections (MTG62)

    Evidence-based recommendations on ClearGuard HD antimicrobial barrier caps for preventing haemodialysis catheter-related bloodstream infections

  14. 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

  15. DyeVert Systems for reducing the risk of acute kidney injury in coronary and peripheral angiography (MTG60)

    Evidence-based recommendations on DyeVert Systems for reducing the risk of acute kidney injury in coronary and peripheral angiography

  16. 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

  17. 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

  18. 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

  19. 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

  20. Parafricta Bootees and Undergarments to reduce skin breakdown in people with or at risk of pressure ulcers (MTG20)

    Evidence-based recommendations on Parafricta Bootees and Undergarments to reduce skin breakdown in people with or at risk of pressure ulcers

  21. WatchBP Home A for opportunistically detecting atrial fibrillation during diagnosis and monitoring of hypertension (MTG13)

    Evidence-based recommendations on the WatchBP Home A for opportunistically detecting atrial fibrillation during diagnosis and monitoring of hypertension

  22. VibraTip for testing vibration perception to detect diabetic peripheral neuropathy (MTG22)

    Evidence-based recommendations on VibraTip for testing vibration perception to detect diabetic peripheral neuropathy