Search results

  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. 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

  4. 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

  5. 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

  6. 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

  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. Fractures (non-complex): assessment and management (NG38)

    This guideline covers assessing and managing non-complex fractures that can be treated in the emergency department or orthopaedic clinic. It aims to improve practice so that people with fractures receive the care that they need without unnecessary tests and treatments.

  10. Myeloma: diagnosis and management (NG35)

    This guideline covers the diagnosing and managing of myeloma (including smouldering myeloma and primary plasma cell leukaemia) in people aged 16 and over. It aims to improve care for people with myeloma by promoting the most effective tests and treatments for myeloma and its complications.

  11. Fractures (complex): assessment and management (NG37)

    This guideline covers assessing and managing pelvic fractures, open fractures and severe ankle fractures (known as pilon fractures and intra-articular distal tibia fractures) in pre-hospital settings (including ambulance services), emergency departments and major trauma centres. It aims to reduce deaths and long-term health problems by improving the quality of emergency and urgent care.