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
Navin Pinto, MD, Professor of Pediatrics at the University of Colorado Anschutz Medical Campus and Colorado Children’s Hospital, discusses the Children’s Oncology Group and their work in neuroblastoma. Neuroblastoma is a rare cancer that largely afflicts...
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)....
