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Abigail Wexner Research Institute, Nationwide Children’s Hospital — Rare Disease Pipeline | Kisho | Kisho

Abigail Wexner Research Institute, Nationwide Children’s Hospital

Other

1

Orphan Designations

0

FDA Approvals

3

Rare Diseases

0

News Articles

Disease	Drug(s)	Designation	Approved
autosomal recessive distal spinal muscular atrophy 1	Single-stranded, non-replicating recombinant adeno-associated viral vector serotype 9 containing the human immunoglobulin u-binding protein 2 cDNA	Orphan Designation	-
spinal muscular atrophy with congenital bone fractures 1	Single-stranded, non-replicating recombinant adeno-associated viral vector serotype 9 containing the human immunoglobulin u-binding protein 2 cDNA	Orphan Designation	-
spinal muscular atrophy with congenital bone fractures 2	Single-stranded, non-replicating recombinant adeno-associated viral vector serotype 9 containing the human immunoglobulin u-binding protein 2 cDNA	Orphan Designation	-

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