by James Radke, PhD | Mar 19, 2026
The US Food and Drug Administration (FDA) has approved Juxtapid (lomitapide) capsules for the treatment of pediatric patients ages 2 years and older with homozygous familial hypercholesterolemia (HoFH). HoFH is a condition characterized by very high levels of...
by Madaline Spencer and James Radke, PhD | Mar 12, 2026
Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses the first-in-human experience of autologous hematopoietic stem cell gene therapy (HSCGT) using a novel ex-vivo lentiviral (LV) platform to correct mucopolysaccharidosis II...
by Madaline Spencer and James Radke, PhD | Mar 11, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the challenges and unmet needs of female patients with Fabry disease. Fabry disease is a type of lysosomal storage disease characterized by deficient alpha-galactosidase...
by James Radke, PhD | Dec 18, 2025
Lisa Abbott, MD, endocrinologist with Northern Nevada Endocrinology, discusses the expansion of broader dosing ranges of Skytrofa (lonapegsomatropin) now available for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD). ...
by James Radke, PhD | 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...
