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Archives for: May 20th, 2019

Dr. Wendy Chung: 2019 Rare Impact Award Honoree

Written by Lisa Sencen on May 20, 2019

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

Orphan Drugs Represent 41 Percent of All New Medications

Written by Jennifer Huron on January 11, 2017

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

Member Spotlight: Cure SMA

Written by Lisa Sencen on August 10, 2015

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