Hemophilia Explained

  Joe Pugliese of the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy...

FDA Approves Scenesse for Erythropoietic Protoporphyria Patients

The U.S. Food and Drug Administration (FDA) granted approval to Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP). For patients who are...

Challenges Facing Rare Disease Clinical Trials

  Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions. Dr. Kenny said there are a myriad of challenges. First among them is the limited number of patients that can qualify for a...

How the Orphan Drug Act Has Changed the Rare Disease Landscape

  The Orphan Drug Act was first signed into law in 1983. The Act provided a variety of incentives for pharmaceutical companies to develop medications specifically for rare diseases, including tax breaks and seven years of market exclusivity. In 1983, there were...
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