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

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

  7. 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

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

    Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

  9. Pressurised intraperitoneal aerosol chemotherapy for peritoneal carcinomatosis (IPG681)

    Evidence-based recommendations on pressurised intraperitoneal aerosol chemotherapy for peritoneal carcinomatosis in adults. This involves spraying the inside of

  10. Endoscopic augmentation of the lower oesophageal sphincter using hydrogel implants for the treatment of gastro-oesophageal reflux disease (IPG222)

    Evidence-based recommendations on endoscopic augmentation of the lower oesophageal sphincter using hydrogel implants for gastro-oesophageal reflux

  11. Arthroscopic knee washout, with or without debridement, for the treatment of osteoarthritis (IPG230)

    Evidence-based recommendations on arthroscopic knee washout, with or without debridement, for treating osteoarthritis

  12. Macular translocation with 360° retinotomy for wet age-related macular degeneration (IPG340)

    Evidence-based recommendations on macular translocation with 360° retinotomy for treating wet (exudative) age-related macular degeneration (AMD)