FDA Puts Hold on Trials for Rare Bone Disease

The U.S. Food and Drug Administration has issued a partial clinical hold in two studies underway to test palovarotene chronic treatment of fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas, respectively. The partial clinical hold is aimed at the...

Testing for Hereditary Angioedema is Easy

  By Peter Ciszewski;  September 30, 2019 Hereditary angioedema (HAE) is a rare genetic disease characterized by repeated edema attacks that can affect any cutaneous or mucosal surface. The most common areas of swelling are the face, larynx, tongue, extremities,...

CRB1 Retinal Disease and Quality of Life

  Kristin Smedley, president of Curing Retinal Blindness Foundation, talks about what doctors need to know about conditions such as retinitis pigmentosa and Leber congenital amaurosis. Curing Retinal Blindness Foundation is a non-profit organization focused on...

Rare Diseases are a Global Concern

  Femida Gwady-Sridhar, PhD, talks her involvement with Rare Disease International (RDI). RDI is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. As Dr. Gwady-Sridhar explains in this...