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

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

  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. 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. 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

  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. Photodynamic therapy for non-melanoma skin tumours (including premalignant and primary non-metastatic skin lesions) (IPG155)

    Evidence-based recommendations on photodynamic therapy for non-melanoma skin tumours (including premalignant and primary non-metastatic skin lesions)

  10. High dose rate brachytherapy in combination with external-beam radiotherapy for localised prostate cancer (IPG174)

    Evidence-based recommendations on high dose rate (HDR) brachytherapy in combination with external-beam radiotherapy (EBRT) for localised prostate cancer

  11. Combined bony and soft tissue reconstruction for hip joint stabilisation in proximal focal femoral deficiency (PFFD) (IPG297)

    Evidence-based recommendations on combined bony and soft tissue reconstruction for hip joint stabilisation in proximal focal femoral deficiency (PFFD)

  12. 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)