Lonafarnib for treating progeria and progeroid laminopathies [ID3867]
Proposed [GID-HST10039] Expected publication date: TBC
Search results
27 results
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VTS-270 for treating Niemann-Pick type C1 (ID1267)
Proposed [GID-HST10020] 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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OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]
Proposed [GID-HST10022] Expected publication date: TBC
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Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]
Proposed [GID-HST10038] 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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Avalglucosidase alfa for treating Pompe disease [ID3737]
Proposed [GID-HST10030] 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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Velmanase alfa for treating alpha-mannosidosis [ID800]
In development [GID-HST10010] 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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Odevixibat for progressive familial intrahepatic cholestasis [ID1570]
In development [GID-HST10043] Expected publication date: 22 January 2022
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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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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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Human alpha1-proteinase inhibitor for treating emphysema ID856
In development [GID-HST10017] Expected publication date: TBC
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Givosiran for treating acute hepatic porphyria [ID1549]
In development [GID-HST10035] Expected publication date: TBC
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Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]
In development [GID-HST10026] Expected publication date: 07 July 2021
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OTL-200 for treating metachromatic leukodystrophy [ID1666]
In development [GID-HST10028] 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: 23 March 2022
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Mental health community settings
In development [GID-SGWAVE0702] Expected publication date: TBC
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Inpatient mental health settings
In development [GID-SGWAVE0701] Expected publication date: TBC
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Management and organisational approaches to safe staffing
In development [GID-SGWAVE0761] Expected publication date: TBC
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Community nursing care settings
In development [GID-SGWAVE0700] Expected publication date: TBC
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Accident and emergency departments
In development [GID-SGWAVE0762] 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