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...
by James Radke, PhD | Nov 21, 2022
Mariana C. Castells MD, PhD, Division of Allergy and Clinical Immunology, Brigham and Women’s Hospital, and Professor of Medicine, Harvard Medical School, gives an overview of mastocytosis. As Dr. Castells explains, mastocytosis is a rare, hematologic...
by James Radke, PhD | Oct 18, 2022
Jennifer McNary, co-founder of One Rare, discusses this innovative new program designed to improve the experiences of young adults with rare diseases. As Ms. McNary explains, a lot of patient advocacy support is aimed toward young children. However, as more...
