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  1. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)

    Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

  2. 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

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

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

  4. Givosiran for treating acute hepatic porphyria (HST16)

    Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over

  5. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

  6. 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

  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. 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

  10. 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

  11. 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

  12. Setmelanotide for treating obesity caused by LEPR or POMC deficiency (HST21)

    Evidence-based recommendations on setmelanotide (Imcivree) for treating obesity caused by LEPR or POMC deficiency in people 6 years and over

  13. Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)

    Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older

  14. Thoracoscopic exclusion of the left atrial appendage (with or without surgical ablation) for non-valvular atrial fibrillation for the prevention of thromboembolism (IPG400)

    Evidence-based recommendations on thoracoscopic exclusion of the left atrial appendage (with or without surgical ablation) for non-valvular atrial fibrillation

  15. 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)

  16. Individually magnetic resonance imaging-designed unicompartmental interpositional implant insertion for osteoarthritis of the knee (IPG317)

    Evidence-based recommendations on inserting individually MRI-designed unicompartmental, interpositional (customised) implant for knee osteoarthritis

  17. Extracorporeal shockwave therapy for refractory plantar fasciitis (IPG311)

    Evidence-based recommendations on extracorporeal shockwave therapy for refractory plantar fasciitis (ligament deterioration between the heel and foot)