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

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

  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. 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

  8. 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

  9. Percutaneous closure of patent foramen ovale for the secondary prevention of recurrent paradoxical embolism in divers (IPG371)

    Evidence-based recommendations on percutaneous closure of patent foramen ovale (PFO) for recurrent neurological decompression sickness in divers

  10. Extraurethral (non-circumferential) retropubic adjustable compression devices for stress urinary incontinence in women (IPG576)

    Evidence-based recommendations on extraurethral (non-circumferential) retropubic adjustable compression devices for stress urinary incontinence in women

  11. Hypoglossal nerve stimulation for moderate to severe obstructive sleep apnoea (IPG598)

    Evidence-based recommendations on hypoglossal nerve stimulation for moderate to severe obstructive sleep apnoea in adults

  12. Artificial heart implantation as a bridge to transplantation for end-stage refractory biventricular heart failure (IPG602)

    Evidence-based recommendations on artificial heart implantation as a bridge to transplantation for end-stage refractory biventricular heart failure

  13. Intramuscular diaphragm stimulation for ventilator-dependent chronic respiratory failure caused by motor neurone disease (IPG593)

    Evidence-based recommendations on intramuscular diaphragm stimulation for ventilator-dependent chronic respiratory failure in people with motor neurone disease

  14. Total distal radioulnar joint replacement for symptomatic joint instability or arthritis (IPG595)

    Evidence-based recommendations on total distal radioulnar joint replacement for symptomatic joint instability or arthritis in adults