by James Radke, PhD | Dec 14, 2022
Guillaume Canaud, MD, PhD, of Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, provides an overview of PiK3CA-related overgrowth syndrome (PROS) and explains why it is important to regularly measure objective outcomes in persons with...
by James Radke, PhD | Dec 12, 2022
Molly Tokaz, MD, from Fred Hutchinson Cancer Center, and Lisa Force, MD, MPH, from University of Washington School of Medicine, discuss the recent analysis of worldwide utilization of hematopoietic stem cell transplantation (HSCT) for the treatment of acute...
by James Radke, PhD | Dec 8, 2022
Pam Vig, PhD, Head of Research & Development at Mirum Pharmaceuticals, gives an overview of Alagille syndrome. As Dr. Vig explains, Alagille syndrome is a genetic disorder that can affect the liver, heart, skeleton, eyes, and kidneys. Liver damage caused...
by James Radke, PhD | Dec 5, 2022
Jeffrey L. Neul, MD, PhD, Director of the Vanderbilt Kennedy Center, and Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, at the Vanderbilt University Medical Center, discusses the need for a multidisciplinary approach to...
by James Radke, PhD | Nov 23, 2022
The U.S. Food and Drug Administration has approved etranacogene dezaparvovec (Hemgenix) for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated serious...
