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 Madaline Spencer | May 31, 2023
Sitra Tauscher-Wisniewski, MD, Vice President of Clinical Development & Analytics at Novartis Gene Therapies, discusses long-term data for onasemnogene abeparvovec (Zolgensma), an FDA-approved gene therapy for spinal muscular atrophy (SMA). SMA is a...
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 Madaline Spencer and James Radke, PhD | May 24, 2023
Shanna Martin, Registered Nurse from Shields Health Solutions, discusses common barriers clinicians face in managing patients with pulmonary arterial hypertension (PAH). PAH is a rare, progressive disorder characterized by high blood pressure (hypertension) in...
by Madaline Spencer and James Radke, PhD | May 23, 2023
Akram Khan, MD, FCCP, Associate Professor of Pulmonary, Allergy, and Critical Care Medicine at Oregon Health & Science University, talks about medical assistance in dying (MAiD) regarding late-stage neuromuscular disorders. Recently, the American College of...
