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1922 results

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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. 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. 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

  5. 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

  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. Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

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

  8. 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

  9. Powered microdebrider turbinoplasty for inferior turbinate hypertrophy (IPG498)

    Evidence-based recommendations on powdered micodebrider turbinoplasty for inferior turbinate hypertrophy (swollen ridges in the nose)

  10. Assessing motility of the gastrointestinal tract using a wireless capsule (IPG502)

    Evidence-based recommendations on assessing motility of the gastrointestinal (GI) tract using a wireless capsule to investigate motility disorders

  11. Faecal microbiota transplant for recurrent Clostridium difficile infection (IPG485)

    Evidence-based recommendations on faecal microbiota (bacteria) transplant for recurrent Clostridium difficile (C. difficile/C. diff) infection

  12. Endoscopic thoracic sympathectomy for primary hyperhidrosis of the upper limb (IPG487)

    Evidence-based recommendations on endoscopic thoracic sympathectomy for primary hyperhidrosis (excess sweating) of the upper limb (palm of hand and armpit)

  13. Chemosaturation via percutaneous hepatic artery perfusion and hepatic vein isolation for primary or metastatic liver cancer (IPG488)

    Evidence-based recommendations on chemosaturation via percutaneous hepatic artery perfusion and hepatic vein isolation for primary or metastatic liver cancer