by Madaline Spencer | Oct 7, 2025
Kevin Eggan, PhD, Chief Scientific Officer at BioMarin, discusses new results from the PEGASUS clinical trial of Palynziq (pegvaliase) for treating patients with phenylketonuria (PKU). PKU is a genetic metabolic disorder that increases the body’s...
by Madaline Spencer | Oct 6, 2025
Amer Zeidan, MBBS, MHS, Professor of Internal Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, discusses plans for a phase 2/3 clinical trial testing bexmarilimab plus standard of care in patients with...
by Madaline Spencer | Oct 3, 2025
Paula Rodriguez Otero, MD, PhD, Hematologist at the University of Navarra, discusses results from the LINKER-SMM1 trial in patients with high-risk smoldering multiple myeloma (HR-SMM). HR-SMM is an asymptomatic clonal plasma cell disorder characterized by a...
by Madaline Spencer | Oct 2, 2025
Clint Allen, MD, Senior Investigator, and Scott Norberg, DO, Associate Research Physician at Center for Cancer Research at the National Cancer Institute, discuss results from a study testing the safety and efficacy of zopapogene imadenovec to treat patients with...
by Madaline Spencer | Oct 1, 2025
Makayla Alger, patient advocate with TUBB4B, and her mother Joann Alger, discuss their experience with the condition and the need for awareness. The TUBB4B gene is a gene believed to be involved in microtubule cytoskeleton organization and the mitotic...
