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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. 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

  3. 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

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

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

  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. 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. Suture fixation of acute disruption of the distal tibiofibular syndesmosis (IPG521)

    Evidence-based recommendations on suture fixation of acute disruption of the distal tibiofibular syndesmosis (ligament injuries)

  10. Intravascular lithotripsy for calcified coronary arteries during percutaneous coronary intervention (IPG673)

    Evidence-based recommendations on intravascular lithotripsy for calcified coronary arteries during percutaneous coronary intervention in adults. This involves

  11. Low-energy contact X-ray brachytherapy (the Papillon technique) for locally advanced rectal cancer (IPG659)

    Evidence-based recommendations on low-energy contact X ray brachytherapy (the Papillon technique) for locally advanced rectal cancer in adults

  12. Short-term circulatory support with left ventricular assist devices as a bridge to cardiac transplantation or recovery (IPG177)

    Evidence-based recommendations on short-term circulatory support with left ventricular assist devices (LVAD) as a bridge to heart transplantation or recovery

  13. High-intensity focused ultrasound for atrial fibrillation in association with other cardiac surgery (IPG184)

    Evidence-based recommendations on high-intensity focused ultrasound for atrial fibrillation (AF) in association with other cardiac surgery

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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