by Peter Ciszewski | Dec 6, 2019
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...
by Peter Ciszewski | Dec 6, 2019
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,...
by Peter Ciszewski | Dec 5, 2019
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...
by Peter Ciszewski | Dec 4, 2019
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...
by Peter Ciszewski | Dec 3, 2019
Andra Stratton, president of Lipodystrophy United explains how lipodystrophy is often misdiagnosed or under-diagnosed. Lipodystrophy is a rare medical condition in which persons have an abnormal distribution of fat in the body. There are numerous types of...
