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  1. 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

  2. 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

  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. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

  5. 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

  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. 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

  10. 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

  11. 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

  12. 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