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OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Proposed [GID-HST10022] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]

Proposed [GID-HST10034] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
VTS-270 for treating Niemann-Pick type C1 (ID1267)

Proposed [GID-HST10020] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Lonafarnib for treating progeria and progeroid laminopathies [ID3867]

Proposed [GID-HST10039] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Arimoclomol for treating Niemann-Pick disease Type C [ID1312]

Proposed [GID-HST10037] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Avalglucosidase alfa for treating Pompe disease [ID3737]

Proposed [GID-HST10030] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Maralixibat for treating type 2 progressive familial intrahepatic cholestasis ID3818

Proposed [GID-HST10040] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed
Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]

Proposed [GID-HST10038] Expected publication date: TBC

Type

Highly specialised technologies guidance

Status

Proposed

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9 results

OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]

VTS-270 for treating Niemann-Pick type C1 (ID1267)

Lonafarnib for treating progeria and progeroid laminopathies [ID3867]

Arimoclomol for treating Niemann-Pick disease Type C [ID1312]

Avalglucosidase alfa for treating Pompe disease [ID3737]

Maralixibat for treating type 2 progressive familial intrahepatic cholestasis ID3818

Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]