A publication at Science Daily recently announced that findings from Children's Hospital of Philadelphia suggested that cysteamine bitartrate, already used for nephropathic cystinosis (a rare kidney disease), might potentially benefit patients…
Jordan Flake and her son Jackson were on their way from El Paso, Texas to Dallas, Texas when they were kicked off their flight because of their Ichthyosis diagnosis. They were on…
Ipsen has just announced that they will be acquiring Clementia Pharmaceuticals. This merging of companies is exciting for the rare community because it will strengthen the resources, knowledge-base, and overall…
According to a story from finanznachrichten.de, the biopharmaceutical company Theranexus has recently released preliminary results from a Phase II clinical trial that is testing the company's experimental product THN102. This…
According to a story from Business Insider, a recent study has found that a team of doctors has a much better chance of making an accurate diagnosis than a single…
According to a story from The Indian Express, a young baby at the Sir Ganga Ram Hospital in Delhi was just diagnosed with an incredibly rare disease that has less…
According to a story from Kractivist, 38 year old Payel Bhattacharya has the rare genetic disorder von Hippel-Lindau disease, or VHL. Payel has written a book about her life called A…
According to a story from Regulatory Affairs Professionals Society, a recent study found that the research and development costs for new molecular entities that were classified as orphan drugs was…
According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the…
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