The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the…
Articles by Mary Chapman
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community…
Due to the current COVID-19 outbreak, Avrobio announced that it has paused dosing new patients in clinical trials…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The…
A Penn State University researcher will use a $65,000 grant from the Lysosomal and Rare Disorders Research and Treatment…
Long before Cerezyme (imiglucerase) and Cerdelga (eliglustat) were approved as Gaucher type 1 treatments, before…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about…
The U.S. Food and Drug Administration (FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases,…