4
Orphan Designations
0
FDA Approvals
4
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Becker muscular dystrophy | Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-muD5) | Orphan Designation | - |
| Duchenne muscular dystrophy | Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-muD5)zildistrogene varoparvovec | Orphan Designation | - |
| Friedreich ataxia | recombinant non-replicating adeno-associated virus serotype hu68 containing a codon-optimized cDNA that encodes the consensus sequence of the human frataxin protein | Orphan Designation | - |
| catecholaminergic polymorphic ventricular tachycardia | adeno-associated viral vector serotype 8 containing the human cardiac calsequestrin gene | Orphan Designation | - |