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
Search results
33 results
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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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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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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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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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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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Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
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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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Migalastat for treating Fabry disease (HST4)
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16
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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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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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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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Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]
In development [GID-HST10026] Expected publication date: TBC
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Ravulizumab for treating paroxysmal nocturnal haemoglobinuria [ID1457]
In development [GID-HST10023] Expected publication date: TBC
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Lysosomal acid lipase deficiency - sebelipase alfa [ID737]
In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] 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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A4250 for treating progressive familial intrahepatic cholestasis [ID1570]
In development [GID-HST10031] 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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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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Metreleptin for treating lipodystrophy (ID861)
In development [GID-HST10011] 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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Avalglucosidase alfa for treating late-onset glycogen storage disease type II [ID3737]
In development [GID-HST10030] 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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Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]
In development [GID-HST10034] 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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Human alpha1-proteinase inhibitor for treating emphysema ID856
In development [GID-HST10017] 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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Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]
In development [GID-HST10015] Expected publication date: TBC
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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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Givosiran for treating acute hepatic porphyria [ID1549]
Proposed [GID-HST10035] 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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Eteplirsen for treating Duchenne muscular dystrophy [ID1003]
In development [GID-HST10007] Expected publication date: TBC
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Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]
In development [GID-HST10004] Expected publication date: TBC