by Victoria Radke | May 28, 2021
The U.S. Food and Drug Administration (FDA) has approved the orphan drug, sotorasib (brand name: Lumakras), to treat adult patients with KRAS G12C-mutated non-small cell lung cancer and who have received at least one prior systemic therapy. This is the first approved...
by Victoria Radke | May 28, 2021
Kathleen Bogart, PhD, Associate Professor of Psychology at Oregon State University, discusses how physicians can help rare disease patients manage their mental health. Dr. Bogart recommends that physicians screen all rare disease patients for mental health as...
by Victoria Radke | May 15, 2021
Happy Huntington Disease Awareness Month! Initiated by the Huntington Society of Canada, Huntington Disease Awareness Month has since become a global movement. Organizations such as the Huntington Disease Society of America and the European Huntington Association...
by Victoria Radke | May 14, 2021
The U.S. Food and Drug Administration (FDA) has approved pegcetacoplan to treat paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, life-threatening blood disorder characterized by hemolysis. Persistently low hemoglobin levels can result in severe fatigue and...
by Victoria Radke | Apr 3, 2021
Endocrine disorders in Prader-Willi syndrome: a model to understand and treat hypothalamic dysfunction In this review, Maithe Tauber and Charlotte Hoyle provide a comprehensive description of the genetics, pathophysiology, and treatment approaches of this rare and...
