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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. 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

  3. 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

  4. 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

  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. 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. 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

  10. 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

  11. 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

  12. 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

  13. 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

  14. 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

  15. Pipeline Flex embolisation device with Shield Technology for the treatment of complex intracranial aneurysms (MTG10)

    Evidence-based recommendations on the Pipeline Flex embolisation device with Shield Technology for the treatment of complex intracranial aneurysms

  16. 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

  17. EXOGEN ultrasound bone healing system for long bone fractures with non-union or delayed healing (MTG12)

    Evidence-based recommendations on EXOGEN ultrasound bone healing system for long bone fractures with non-union or delayed healing