1
Orphan Designations
0
FDA Approvals
1
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Charcot-Marie-Tooth disease type 1A | a non-replicating recombinant adeno- associated virus serotype 9 (AAV9) based gene therapy vector containing the DNA of Streptococcus pyogenes Cas9 (SpCas9) protein and single guide RNA (sgRNA) designed to target the TATA-box of the PMP22 P1 promoter | Orphan Designation | - |