As a clinical and molecular geneticist, Wendy Chung, M.D., Ph.D. has been working with patients with rare genetic diseases and their families for the last 20 years. She is the Kennedy Family Professor of Pediatrics and Medicine at Columbia University Irving Medical Center and leader of the Chung… Read More
Washington, D.C., January 11, 2017—Data released by the U.S. Food and Drug Administration (FDA) show that 9 of the 22 new medications approved in 2016, or 41 percent, are orphan drugs to treat rare diseases.
“The percentage of orphan drugs approved in recent years by FDA remains high and this is welcome news for the 30… Read More
The Hill, a top U.S. political website, read by Congressional offices, the White House, political pundits, and more, today published the following op-ed by NORD President and CEO, Peter L. Saltonstall, urging Congress to reauthorize the Rare Pediatric Disease Priority… Read More
Cure SMA is a voluntary-driven, non-profit organization dedicated solely to eradicating SMA by promoting and supporting research, helping families cope through informational programs and support, and educating the public and the medical community about SMA.
1.) What does it mean to you personally to be a patient organization serving the rare… Read More
