Therapeutics
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| Name | Synonyms | FDA Status | Company | Target Type | Therapy Type | Approved For |
|---|---|---|---|---|---|---|
| Arimoclomol | MiplyffaTM | Niemann-Pick Diseases (Approved), Amyotrophic Lateral Sclerosis (Discontinued), Gaucher Disease (Discontinued) | Zevra Therapeutics | Cholesterol, Other | Small Molecule | Niemann-Pick Disease Type C |