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National Institutes of Health National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) — Rare Disease Pipeline | Kisho | Kisho

National Institutes of Health National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS)

Other

Orphan Designations

FDA Approvals

Rare Diseases

News Articles

Disease	Drug(s)	Designation	Approved
autosomal recessive leukoencephalopathy-ischemic stroke-retinitis pigmentosa syndrome	recombinant adeno-associated virus 5 vector expressing a functional human cyclic nucleotide gated channel subunit beta 1 (CNGB1) gene	Orphan Designation	-
posterior column ataxia-retinitis pigmentosa syndrome	recombinant adeno-associated virus 5 vector expressing a functional human cyclic nucleotide gated channel subunit beta 1 (CNGB1) gene	Orphan Designation	-
retinitis pigmentosa 4	recombinant adeno-associated virus 5 vector expressing a functional human cyclic nucleotide gated channel subunit beta 1 (CNGB1) gene	Orphan Designation	-

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