As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
Within the last week, biotechnology company Levo Therapeutics announced results from the Phase 3 CARE-PWS clinical trial. During the trial, researchers examined the efficacy, safety, and tolerability of LV-101…
The FDA recently granted Orphan Drug status to CS1, a drug therapy from Cereno Scientific, according to Pulmonary Hypertension News. CS1 is designed to provide safe and effective treatment for…
As originally reported in Porphyria News, rare diseases have historically been neglected by traditional medicine; their obscure and often genetic nature made them lack priority, stuck behind a long list…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
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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