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...
by Madaline Spencer | 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 Madaline Spencer | Sep 30, 2025
Krista Casazza, PhD, co-author of the recent article “Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment” in the Journal of Inherited and Metabolic Disease, discusses biomarker validation in Niemann-Pick disease type C (NPC1)....
