Leriglitazone for treating andrenoleukodystrophy [ID3903]
Awaiting development [GID-HST10049] Expected publication date: TBC
Search results
6 results
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OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]
Awaiting development [GID-HST10022] Expected publication date: TBC
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Lonafarnib for treating progeria and progeroid laminopathies [ID3867]
Awaiting development [GID-HST10039] Expected publication date: TBC
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VTS-270 for treating Niemann-Pick type C1 (ID1267)
Awaiting development [GID-HST10020] Expected publication date: TBC
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Asfotase alfa for treating paediatric-onset hypophosphatasia (review of HST6) [ID3927]
Awaiting development [GID-HST10046] Expected publication date: 15 December 2022
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Palovarotene for preventing heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID3739]
Awaiting development [GID-HST10032] Expected publication date: TBC