Bioworld.com

Washington, D.C.—Senators Orrin Hatch, R-Utah, and Amy Klobuchar, D-Minn., the co-chairs of the Senate Rare Disease Caucus, participated in a colloquy this afternoon to discuss how best to incorporate the patient voice into broader policy conversations...

Who’s “cute, red-headed, and freckle faced?” It’s Gabe! The 11-year-old has been following three cyclists in their 2,200 mile bike ride from Maine to Alabama, and Gabe’s star power is rising. On Day 5 of the 9-day ride, Gabe’s team pedaled through Buffalo...

There’s a natural tension between wanting the FDA to ensure safety and efficacy before a drug enters the market and wanting to speed up what many view as a glacially slow approval process. The rare disease community tends to fall in the second camp,...

In 2007, Congress created a voucher program that shaves months off of the Food and Drug Administration’s review process for drug development that targets rare pediatric diseases. Photo by Getty Images Drugmaker Sarepta Therapeutics won a big victory...

I met a young boy named Gabe Griffin a few years back at Battleship Park in Mobile. His family and friends had organized a cross country bike ride that started in Oregon and ended on Alabama’s Gulf Coast. The goal of the ride was to raise awareness...