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  1. 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

  2. Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)

    Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

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

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

  4. 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

  5. Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)

    Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older

  6. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children

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

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

  10. 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

  11. 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

  12. 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

  13. Faecal microbiota transplant for recurrent Clostridium difficile infection (IPG485)

    Evidence-based recommendations on faecal microbiota transplant for recurrent Clostridium difficile infection. This involves introducing enteric bacteria from

  14. Ultrasound-guided percutaneous radiofrequency ablation for benign thyroid nodules (IPG562)

    Evidence-based recommendations on ultrasound-guided percutaneous radiofrequency ablation for treating benign thyroid nodules in adults

  15. Low-intensity pulsed ultrasound to promote healing of fresh fractures at high risk of non-healing (IPG622)

    Evidence-based recommendations on low-intensity pulsed ultrasound to promote healing of fresh fractures at high risk of non-healing in adults. This involves

  16. Low-intensity pulsed ultrasound to promote healing of delayed-union and non-union fractures (IPG623)

    Evidence-based recommendations on low-intensity pulsed ultrasound to promote healing of delayed-union and non-union fractures in adults. This involves using an

  17. Transurethral water vapour ablation for lower urinary tract symptoms caused by benign prostatic hyperplasia (IPG625)

    Evidence-based recommendations on transurethral water vapour ablation for lower urinary tract symptoms caused by benign prostatic hyperplasia in adults

  18. Transcutaneous neuromuscular electrical stimulation for oropharyngeal dysphagia in adults (IPG634)

    Evidence-based recommendations on transcutaneous neuromuscular electrical stimulation for oropharyngeal dysphagia in adults. This involves electrically

  19. Transurethral water jet ablation for lower urinary tract symptoms caused by benign prostatic hyperplasia (IPG629)

    Evidence-based recommendations on transurethral water jet ablation for lower urinary tract symptoms caused by benign prostatic hyperplasia in adults

  20. Selective internal radiation therapy for unresectable primary intrahepatic cholangiocarcinoma (IPG630)

    Evidence-based recommendations on selective internal radiation therapy for unresectable primary intrahepatic cholangiocarcinoma in adults. This involves

  21. Percutaneous insertion of a temporary heart pump for left ventricular haemodynamic support in high-risk percutaneous coronary interventions (IPG633)

    Evidence-based recommendations on percutaneous insertion of a temporary heart pump for left ventricular haemodynamic support in high-risk percutaneous coronary

  22. Prostate artery embolisation for lower urinary tract symptoms caused by benign prostatic hyperplasia (IPG611)

    Evidence-based recommendations on prostate artery embolisation for lower urinary tract symptoms caused by benign prostatic hyperplasia in adults