by Madaline Spencer and James Radke, PhD | May 22, 2026
Pablo Villoslada, MD, PhD, Founder and Medical Director of Accure Therapeutics and Head of Pathogenesis and New Therapies MS at IDIBAPS in Hospital Clínic in Barcelona, discusses results from the ACUITY clinical trial testing privosegtor to treat patients with acute...
by James Radke, PhD | May 14, 2026
Richard J. Auchus, MD, PhD, Professor of Internal Medicine and Pharmacology at the University of Michigan Medical School, discusses two-year results of Crenessity (crinecerfont) in the treatment of congenital adrenal hyperplasia (CAH). CAH refers to a group of...
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...
