by James Radke, PhD | Dec 18, 2025
Lisa Abbott, MD, endocrinologist with Northern Nevada Endocrinology, discusses the expansion of broader dosing ranges of Skytrofa (lonapegsomatropin) now available for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). ...
by James Radke, PhD | Sep 30, 2025
The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for the treatment of children ages 1 to 5 years old with homozygous familial hypercholesterolemia (HoFH), supplemental to diet, exercise, and other lipid-lowering therapies. HoFH is a...
by James Radke, PhD | Aug 1, 2025
Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their organizations’ collective studies showing the direct and indirect...
by James Radke, PhD | Aug 1, 2025
Henry J. Kaminski, MD, Professor of Medicine at The George Washington University describes the value of being in the Rare Diseases Clinical Research Network (RDCRN). Dr. Kaminsky leads the Dr. Kaminski leads the Myasthenia Gravis Rare Disease Network (MGNET), a...
by James Radke, PhD | Jul 3, 2025
Stephanie Davis, MD, and Thomas Ferkol, MD, Professors of Medicine at University of North Carolina at Chapel Hill, discuss the value of being part of the Rare Diseases Clinical Research Network (RDCRN). Drs. Davis and Ferkol are co-leaders of the Genetic Disorders...
