5
Orphan Designations
0
FDA Approvals
18
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| HELLP syndrome | Vyjuvek | Orphan Designation | - |
| Hailey-Hailey disease | a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector engineered to express full-length human ATPase Secretory Pathway Calcium Transporting 1 (ATP2C1) | Orphan Designation | - |
| Tangier disease | A replication-incompetent, non-integrating Herpes simplex type 1 (HSV-1) vector expressing the human transglutaminase-1 (TGM-1) enzyme | Orphan Designation | - |
| acral dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| alpha 1-antitrypsin deficiency | A replication-defective, non-integrating herpes simplex virus type 1-based vector engineered to express full-length, functional human alpha-1 antitrypsin | Orphan Designation | - |
| autosomal recessive congenital ichthyosis | A replication-incompetent, non-integrating Herpes simplex type 1 (HSV-1) vector expressing the human transglutaminase-1 (TGM-1) enzyme | Orphan Designation | - |
| autosomal recessive congenital ichthyosis 1 | A replication-incompetent, non-integrating Herpes simplex type 1 (HSV-1) vector expressing the human transglutaminase-1 (TGM-1) enzyme | Orphan Designation | - |
| centripetalis recessive dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| cystic fibrosis | A replication-defective, non-integrating herpes simplex virus type 1 vector expressing human cystic fibrosis transmembrane conductance regulator | Orphan Designation | - |
| dystrophic epidermolysis bullosa pruriginosa | Vyjuvek | Orphan Designation | - |
| dystrophic epidermolysis bullosa, nails only | Vyjuvek | Orphan Designation | - |
| generalized dominant dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| hairy tongue | a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector engineered to express full-length human ATPase Secretory Pathway Calcium Transporting 1 (ATP2C1) | Orphan Designation | - |
| localized dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| oral hairy leukoplakia | a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector engineered to express full-length human ATPase Secretory Pathway Calcium Transporting 1 (ATP2C1) | Orphan Designation | - |
| pretibial dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| recessive dystrophic epidermolysis bullosa | Vyjuvek | Orphan Designation | - |
| recessive dystrophic epidermolysis bullosa-generalized other | Vyjuvek | Orphan Designation | - |