2
Orphan Designations
0
FDA Approvals
9
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Aicardi syndrome | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |
| COACH syndrome | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |
| TUBB2A-related tubulinopathy | Antisense oligonucleotide targeting TUBB4A mRNA | Orphan Designation | - |
| TUBB4A-related neurologic disorder | Antisense oligonucleotide targeting TUBB4A mRNA | Orphan Designation | - |
| early-onset progressive diffuse brain atrophy-microcephaly-muscle weakness-optic atrophy syndrome | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |
| fucosidosis | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |
| hypomyelinating leukodystrophy 6 | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |
| macrothrombocytopenia, isolated, 1, autosomal dominant | Antisense oligonucleotide targeting TUBB4A mRNA | Orphan Designation | - |
| neurodevelopmental disorder with microcephaly, epilepsy, and hypomyelination | single stranded, synthetic, hybrid RNA/DNA antisense oligonucleotide (ASO) gapmer targeting the TUBB4A gene | Orphan Designation | - |