As originally reported in CheckOrphan, there's good news today for adult patients with acute hepatic porphyria, a genetic disorder which causes pain and paralysis. The Food and Drug Administration (FDA)…
According to an article published on BioPortfolio, earlier this week, biotechnology company Vybion Incorporated secured orphan drug designation for its highly experimental Huntington's disease drug INT41. About Huntington's Disease Huntington's…
According to an article published by Biospace, X4 Pharmaceuticals, based in Cambridge, Massachusetts, has recently announced successful phase two results in clinical trials for their experimental drug X4P-001. The drug…
If drugs were measured by the quantity of approvals, orphan drugs would make quite a splash. Measured in more financial terms, however, they make up a much smaller portion of…
Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G). According to the FDA, the Orphan Drug Designation program provides…
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