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
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)....
by Madaline Spencer | Sep 29, 2025
The U.S. Food and Drug Administration (FDA) has approved Palsonify (paltusotine) for the first-line treatment of adults with acromegaly with inadequate response to surgery and/or for whom surgery is not an option. Acromegaly is a hormonal disorder that results from...
by Madaline Spencer | Sep 29, 2025
Jamas LaFreniere, Founder of Sophie’s Hope Foundation and CURE GSD1b, and Blair Stone-Schneider, Executive Director of Sophie’s Hope Foundation, discuss their patient advocacy organization and starting a natural history study for glycogen storage disease type 1b...
