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Archives for: December 12th, 2019

CME: Spinal Muscular Atrophy

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

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