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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. Procalcitonin testing for diagnosing and monitoring sepsis (ADVIA Centaur BRAHMS PCT assay, BRAHMS PCT Sensitive Kryptor assay, Elecsys BRAHMS PCT assay, LIAISON BRAHMS PCT assay and VIDAS BRAHMS PCT assay) (DG18)

    Evidence-based recommendations on procalcitonin testing for diagnosing and monitoring sepsis (ADVIA Centaur BRAHMS PCT assay, BRAHMS PCT Sensitive

  14. SepsiTest assay for rapidly identifying bloodstream bacteria and fungi (DG20)

    Evidence-based recommendations on the SepsiTest assay for rapidly identifying bloodstream bacteria and fungi

  15. Integrated sensor-augmented pump therapy systems for managing blood glucose levels in type 1 diabetes (the MiniMed Paradigm Veo system and the Vibe and G4 PLATINUM CGM system) (DG21)

    Evidence-based recommendations on the MiniMed Paradigm Veo and Vibe and G4 PLATINUM CGM sensor-augmented pump therapy systems, which combine continuous glucose

  16. PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA Xpress PlGF 1-2-3 test, and BRAHMS sFlt-1 Kryptor/BRAHMS PlGF plus Kryptor PE ratio) (DG23)

    Evidence-based recommendations on PlGF-based testing to help diagnose suspected pre-eclampsia (Triage PlGF test, Elecsys immunoassay sFlt-1/PlGF ratio, DELFIA

  17. EarlyCDT Lung for assessing risk of lung cancer in solid lung nodules (DG46)

    Evidence-based recommendations on EarlyCDT Lung for assessing the risk of lung cancer in solid lung nodules

  18. PredictSURE IBD and IBDX to guide treatment of Crohn's disease (DG45)

    Evidence-based recommendations on PredictSURE IBD and IBDX for guiding treatment of Crohn’s disease

  19. Integrated multiplex PCR tests for identifying gastrointestinal pathogens in people with suspected gastroenteritis (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B assay) (DG26)

    Evidence-based recommendations on integrated multiplex polymerase chain tests (xTAG Gastrointestinal Pathogen Panel, FilmArray GI Panel and Faecal Pathogens B

  20. Quantitative faecal immunochemical tests to guide referral for colorectal cancer in primary care (DG30)

    Evidence-based recommendations on faecal immunochemical tests (OC Sensor, HM-JACKarc, FOB Gold and RIDASCREEN) to guide GP referral for colorectal cancer

  21. Tests in secondary care to identify people at high risk of ovarian cancer (DG31)

    Evidence-based recommendations on tests in secondary care to identify people at high risk of ovarian cancer. The tests are the IOTA ADNEX model, Overa (MIA2G)

  22. Adjunctive colposcopy technologies for assessing suspected cervical abnormalities: the DYSIS colposcope with DYSISmap and the ZedScan I (DG32)

    Evidence-based recommendations on 2 adjunctive colposcopy technologies (the DYSIS colposcope with DYSISmap and the ZedScan I) for assessing suspected cervical

  23. Faecal calprotectin diagnostic tests for inflammatory diseases of the bowel (DG11)

    Evidence-based recommendations on faecal calprotectin tests for distinguishing between inflammatory bowel diseases (such as Crohn’s disease and ulcerative

  24. Measuring fractional exhaled nitric oxide concentration in asthma: NIOX MINO, NIOX VERO and NObreath (DG12)

    Evidence-based recommendations on NIOX MINO, NIOX VERO and NObreath for measuring the amount of exhaled nitric oxide (FeNO) in the breath to help diagnose

  25. High-sensitivity troponin tests for the early rule out of NSTEMI (DG40)

    Evidence-based recommendations on high-sensitivity troponin tests for the early rule out of NSTEMI (non-ST-segment elevation myocardial infarction)

  26. Implantable cardiac monitors to detect atrial fibrillation after cryptogenic stroke (DG41)

    Evidence-based recommendations on implantable cardiac monitors to detect atrial fibrillation after cryptogenic stroke

  27. QAngio XA 3D QFR and CAAS vFFR imaging software for assessing coronary stenosis during invasive coronary angiography (DG43)

    Evidence-based recommendations on QAngio XA 3D QFR and CAAS vFFR imaging software for assessing coronary stenosis during invasive coronary angiography

  28. Tests to help assess risk of acute kidney injury for people being considered for critical care admission (ARCHITECT and Alinity i Urine NGAL assays, BioPorto NGAL test and NephroCheck test) (DG39)

    Evidence-based recommendations on tests to help assess risk of acute kidney injury for people being considered for critical care admission. The tests are the

  29. Atrial fibrillation and heart valve disease: self-monitoring coagulation status using point-of-care coagulometers (the CoaguChek XS system) (DG14)

    Evidence-based recommendations on the CoaguChek XS system for self-monitoring coagulation status in adults and children. The recommendations originally included

  30. Molecular testing strategies for Lynch syndrome in people with colorectal cancer (DG27)

    Evidence-based recommendations on using immunohistochemistry or microsatellite instability testing to guide further testing for Lynch syndrome in people with

  31. SeHCAT (tauroselcholic [75 selenium] acid) for diagnosing bile acid diarrhoea (DG44)

    Evidence-based recommendations on SeHCAT (tauroselcholic [75 selenium] acid) for diagnosing bile acid diarrhoea

  32. Rapid tests for group A streptococcal infections in people with a sore throat (DG38)

    Evidence-based recommendations on rapid tests for group A streptococcal infections in people aged 5 and over with a sore throat

  33. Lead-I ECG devices for detecting symptomatic atrial fibrillation using single time point testing in primary care (DG35)

    Evidence-based recommendations on lead-I electrocardiogram (ECG) devices (imPulse, Kardia Mobile, MyDiagnostick and Zenicor-ECG) for detecting symptomatic

  34. SonoVue (sulphur hexafluoride microbubbles) – contrast agent for contrast-enhanced ultrasound imaging of the liver (DG5)

    Evidence-based recommendations on SonoVue (sulphur hexafluoride microbubbles) for contrast-enhanced ultrasound imaging of the liver