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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. 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

  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. 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

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

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

  9. 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

  10. Percutaneous endovascular forearm arteriovenous fistula creation for haemodialysis access (IPG710)

    Evidence-based recommendations on percutaneous endovascular forearm arteriovenous fistula creation for haemodialysis access in adults. This involves using

  11. Self-expanding implant insertion into the intersphincteric space for faecal incontinence (IPG685)

    Evidence-based recommendations on self-expanding implant insertion into the intersphincteric space for faecal incontinence. This involves inserting implants

  12. Melphalan chemosaturation with percutaneous hepatic artery perfusion and hepatic vein isolation for primary or metastatic cancer in the liver (IPG691)

    Evidence-based recommendations on melphalan chemosaturation with percutaneous hepatic artery perfusion and hepatic vein isolation for primary or metastatic canc

  13. Percutaneous insertion of a closure device to repair a paravalvular leak around a replaced mitral or aortic valve (IPG700)

    Evidence-based recommendations on percutaneous insertion of a closure device to repair a paravalvular leak around a replaced mitral or aortic valve. This involv

  14. Balloon dilatation with or without stenting for pulmonary artery or non-valvar right ventricular outflow tract obstruction in children (IPG76)

    Evidence-based recommendations on balloon dilatation for pulmonary artery or non-valvar right ventricular outflow tract obstruction in children

  15. Deep brain stimulation for chronic, severe, treatment-resistant obsessive-compulsive disorder in adults (IPG693)

    Evidence-based recommendations on deep brain stimulation for chronic, severe, treatment-resistant obsessive-compulsive disorder in adults. This involves implant

  16. Ex-situ machine perfusion for extracorporeal preservation of lungs (ex-vivo lung perfusion) for transplant (IPG695)

    Evidence-based recommendations on ex-situ machine perfusion for extracorporeal preservation of lungs for transplant. This involves using a machine to deliver

  17. Inducing and maintaining normothermia using temperature modulation devices to improve outcomes after stroke or subarachnoid haemorrhage (IPG701)

    Evidence-based recommendations on inducing and maintaining normothermia using temperature modulation devices after stroke or subarachnoid haemorrhage in adults

  18. 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