Written by Valaree DonFrancesco on December 12, 2019
Spinal Muscular Atrophy: Best Practices in Diagnosis and Management is a live educational webinar that will summarize clinical practice and consensus guidelines for the comprehensive management of spinal muscular atrophy (SMA). Strategies for incorporating new disease-modifying therapies for SMA into daily clinical practice will be highlighted.
This webinar is intended for pediatric neurologists, neurologists,… Read More
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
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
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