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  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. 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

  10. 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

  11. 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

  12. 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