by Madaline Spencer and James Radke, PhD | Jun 12, 2023
Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains why it often takes years to properly diagnose Primary Immunodeficiency (PI). PI is a heterogeneous group of inherited disorders characterized by impaired immune...
by Craig McCabe and James Radke, PhD | Jun 5, 2023
Rakesh Jain, MD, MPH, Professor, Department of Psychiatry, Texas Tech University School of Medicine – Permian Basin, discusses the recent Huntington’s disease treatment approval. Newly approved deutetrabenazine (Austedo XR) extended-release tablets...
by Craig McCabe and James Radke, PhD | Jun 1, 2023
Jack Johnson, of the Fabry Support and Information Group (FSIG) talks about the different types of pain often associated with Fabry disease. Fabry disease is a rare, X-linked lysosomal storage disorder that is caused by the deficiency of the enzyme...
by Madaline Spencer and James Radke, PhD | May 30, 2023
Samiah Al-Zaidy, MD, Vice President of Clinical Development and Lead on the Passage Bio GM1 Gangliosidosis (GM1) Program, gives an overview of GM1. As Dr. Al-Zaidy explains, GM1 is an inherited lysosomal disorder caused by mutations in the GLB1 gene. These...
by James Radke, PhD | May 29, 2023
Kerry Mello-Parker, Director of Clinical Compliance for Rare & Complex Therapies, and Shanna Martin, Registered Nurse, from Shields Health Solutions, discuss the Risk Evaluation and Mitigation Strategies (REMS) program for macitentan, a treatment option for...
