VTS-270 for treating Niemann-Pick type C1 (ID1267)
Proposed [GID-HST10020] Expected publication date: TBC
Search results
10 results
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Setmelanotide for treating obesity caused by LEPR or POMC deficiency [ID3764]
Proposed [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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Maralixibat for treating type 2 progressive familial intrahepatic cholestasis ID3818
Proposed [GID-HST10040] 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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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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Lonafarnib for treating progeria and progeroid laminopathies [ID3867]
Proposed [GID-HST10039] 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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Odevixibat (A 4250) for progressive familial intrahepatic cholestasis [ID1570]
Proposed [GID-HST10043] Expected publication date: TBC