Raring to go for Rare Disease Day

Blue-Jeans-Denim-Ribbon-Rare-Disease.jpg

Scientists are in rare form today, donning denim ribbons in support of rare diseases today. Introduced in 2008, Rare Disease Day aims to raise awareness for orphan diseases that often fall under the radar. Keeping with this mission, below we highlight some of Nature Medicine’s recent coverage into issues faced by rare disease researchers.

Last month, we reported about Sanofi-Aventis’ takeover of Genzyme Corporation, the Cambridge, Massachusetts-based biotech that made its name by developing therapies for Gaucher disease, Fabry disease and other rare disorders. But buy-outs are not the only way that big pharma is getting in the rare disease game. In August 2010, we highlighted the growing trend of drug makers launching new research units devoted exclusively to developing and commercializing new drugs targeting rare diseases. Also in August, we reported on new ways that patient advocacy groups are trying to raise funds for research and clinical trials for rare disease therapeutics

Despite all these developments, however, approvals for new orphan drugs remain frustratingly low. In a story posted on our blog earlier today (and in the upcoming March issue of Nature Medicine), we note that orphan drug designations are at an all-time high, yet the number given the go ahead by federal regulators lags behind.

Leave a Reply

Your email address will not be published. Required fields are marked *