4
Orphan Designations
0
FDA Approvals
7
Rare Diseases
3
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | phosphoryl guanidine-modified stereopure splicing antisense oligonucleotide against DMD mRNA inducing exon 53 skippingSynthetic stereopure antisense oligonucleotide specific for human dystrophin pre-messenger ribonucleic acid (mRNA) | Orphan Designation | - |
| Huntington disease | synthetic stereopure antisense oligonucleotide specific to the mutant huntingtin mRNA transcript at the U variant of single nucleotide polymorphism rs326331antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |
| Huntington disease-like 1 | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |
| Huntington disease-like 2 | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |
| Huntington disease-like 3 | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |
| Westphal disease | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |
| malignancy diagnosed during pregnancy | antisense oligonucleotide targeting the U isoform of SNP rs362307 | Orphan Designation | - |