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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. 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. 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

  4. 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

  5. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

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

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia 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. 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

  9. 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

  10. 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

  11. 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

  12. The Sherlock 3CG Tip Confirmation System for placement of peripherally inserted central catheters (MTG24)

    Evidence-based recommendations on The Sherlock 3CG Tip Confirmation System for placement of peripherally inserted central catheters

  13. 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

  14. 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

  15. 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

  16. UroLift for treating lower urinary tract symptoms of benign prostatic hyperplasia (MTG26)

    Evidence-based recommendations on UroLift for treating lower urinary tract symptoms of benign prostatic hyperplasia

  17. Tegaderm CHG securement dressing for vascular access sites in critically ill adults (MTG25)

    Evidence-based recommendations on The 3M Tegaderm CHG IV securement dressing for central venous and arterial catheter insertion sites

  18. 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