3
Orphan Designations
0
FDA Approvals
5
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| RYR1-related myopathy | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Orphan Designation | - |
| carbon monoxide-induced delayed encephalopathy | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Orphan Designation | - |
| catecholaminergic polymorphic ventricular tachycardia | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Orphan Designation | - |
| multisystemic smooth muscle dysfunction syndrome | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Orphan Designation | - |
| natal teeth-intestinal pseudoobstruction-patent ductus syndrome | 4-[(7-Methoxy-2,3-dihydro-1,4-benzothiazepin-4(5H)-yl)methyl]benzoic acid, hemifumarate | Orphan Designation | - |