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...
by Peter Ciszewski | Oct 17, 2022
Pam Vig, PhD, Head of Research & Development at Mirum Pharmaceuticals, discusses how the approval of maralixibat has changed the management of Alagille syndrome. Alagille syndrome is a genetic disorder that can affect the liver, heart, skeleton, eyes, and...
by Peter Ciszewski | Oct 13, 2022
Ajay K. Nooka, MD, Associate Professor of Hematology and Medical Oncology at Emory School of Medicine, discusses the current treatment landscape for multiple myeloma. Multiple myeloma is a rare blood cancer associated with uncontrolled growth of plasma cells....
by Peter Ciszewski | Oct 11, 2022
Bernd Kasper, MD, PhD, University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the phase 3 DeFi clinical trial, discusses the impressive results of the trial, which evaluated nirogacestat in the treatment of desmoid...
by Peter Ciszewski | Oct 10, 2022
Olaf Bodamer, MD, PhD, Associate Chief of Genetics and Genomics at Boston Children’s Hospital, discusses his background. As Dr. Bodamer explains, he started his career as a pediatrician, specializing in metabolism. His interest in genetic metabolic...
