Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
Search results
1515 results
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Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency
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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
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Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies
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Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa in adults and children
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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
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Migalastat for treating Fabry disease (HST4)
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
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Eliglustat for treating type 1 Gaucher disease (HST5)
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
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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
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Volanesorsen for treating familial chylomicronaemia syndrome (HST13)
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults
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Metreleptin for treating lipodystrophy (HST14)
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults
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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
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Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children
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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
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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
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OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]
Proposed [GID-HST10022] Expected publication date: TBC
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Human alpha1-proteinase inhibitor for treating emphysema ID856
In development [GID-HST10017] Expected publication date: TBC
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Maralixibat for treating type 2 progressive familial intrahepatic cholestasis ID3818
Proposed [GID-HST10040] Expected publication date: TBC
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Palovarotene for treating flare ups of heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID2721]
In development [GID-HST10033] Expected publication date: TBC
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Palovarotene for preventing heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID3739]
In development [GID-HST10032] Expected publication date: TBC
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VTS-270 for treating Niemann-Pick type C1 (ID1267)
Proposed [GID-HST10020] Expected publication date: TBC
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Avalglucosidase alfa for treating Pompe disease [ID3737]
Proposed [GID-HST10030] Expected publication date: TBC
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Afamelanotide for treating erythropoietic protoporphyria [ID927]
In development [GID-HST10009] Expected publication date: TBC
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Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223
Proposed [GID-HST10019] Expected publication date: TBC
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Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]
In development [GID-HST10027] Expected publication date: TBC
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Velmanase alfa for treating alpha-mannosidosis [ID800]
In development [GID-HST10010] Expected publication date: TBC
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Lonafarnib for treating progeria and progeroid laminopathies [ID3867]
Proposed [GID-HST10039] Expected publication date: TBC
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Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 years and over [ID1590]
In development [GID-HST10045] Expected publication date: 05 May 2022
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Givosiran for treating acute hepatic porphyria [ID1549]
In development [GID-HST10035] Expected publication date: TBC
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Arimoclomol for treating Niemann-Pick disease Type C [ID1312]
Proposed [GID-HST10037] Expected publication date: TBC
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Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (review of HST3) ID1642
In development [GID-HST10044] Expected publication date: TBC
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Asfotase alfa for treating paediatric-onset hypophosphatasia (review of HST6) [ID3927]
Proposed [GID-HST10046] Expected publication date: TBC
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OTL-200 for treating metachromatic leukodystrophy [ID1666]
In development [GID-HST10028] Expected publication date: TBC
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OTL-200 for treating metachromatic leukodystrophy [ID1666]: evaluation consultation
We are listening to your views on this highly specialised technology. Comments close 30 July 2021.
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Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]
In development [GID-HST10034] Expected publication date: 23 March 2022
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Odevixibat for progressive familial intrahepatic cholestasis [ID1570]
In development [GID-HST10043] Expected publication date: 26 January 2022
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Lysosomal acid lipase deficiency - sebelipase alfa [ID737]
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC
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Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]
In development [GID-HST10038] Expected publication date: TBC
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EarlyCDT Lung for lung cancer risk classification of solid pulmonary nodules
In development [GID-DG10041] Expected publication date: 23 February 2022
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PredictSURE IBD and IBDX to guide treatment of Crohn's disease
In development [GID-DG10029] Expected publication date: TBC
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Transperineal biopsy in people with suspected prostate cancer
In development [GID-DG10043] Expected publication date: 22 June 2022
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Software with artificial intelligence derived algorithms for analysing CT brain scans in people with a suspected acute stroke
In development [GID-DG10044] Expected publication date: 20 July 2022
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LiverMultiScan MRI imaging software for the diagnosis and characterisation of non-alcoholic fatty liver disease (provisional title)
In development [GID-DG10045] Expected publication date: 26 October 2022
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PlGF-based testing to help diagnose suspected pre-eclampsia (update of DG23)
In development [GID-DG10040] Expected publication date: TBC
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Exploratory economic modelling of SARS-CoV-2 viral detection point of care tests and serology tests
In development [GID-DG10038] Expected publication date: TBC
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Quantitative faecal immunochemical tests to guide colorectal cancer pathway referral for people with a change in bowel habit or abdominal pain
In development [GID-DG10036] Expected publication date: TBC
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The EOS 2D/3D imaging system (DG1)
Evidence-based recommendations on the EOS 2D/3D imaging system for producing 2D and 3D images of the skeleton
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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
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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
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VivaScope 1500 and 3000 imaging systems for detecting skin cancer lesions (DG19)
Evidence-based recommendations on the VivaScope 1500 and 3000 imaging systems for detecting skin cancer lesions