by Madaline Spencer | Jul 24, 2025
Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. Fabry disease is a rare lysosomal storage disease characterized by a deficiency in the enzyme alpha-galactosidase (alpha-GAL). Alpha-GAL...
by Madaline Spencer | Jul 23, 2025
Timothy Craig, DO, Tenured Professor of Medicine, Pediatrics, and Biomedical Sciences at Penn State University, Principal Investigator of the VANGUARD trial, discusses the recent approval of Andembry (garadacimab) for patients ages 12 years and older with hereditary...
by Madaline Spencer | Jul 22, 2025
Matthias Löhr, MD, PhD, Professor of Gastroenterology & Hepatology at Karolinska Institutet, Stockholm, Sweden, discusses recent data from the MITIGATE clinical trial of inebilizumab for the treatment of IgG4-related disease (IgG4-RD). IgG4-RD is an...
by Madaline Spencer | Jul 21, 2025
Amélie Lothe, PhD, Head of Global Medical Community of Rare Epilepsies at UCB, discusses positive data on fenfluramine for the treatment of CDKL5 deficiency disorder (CDD). CDD is a rare, genetic developmental and epileptic encephalopathy (DEE)...
by Madaline Spencer | Jul 18, 2025
Sandra Silvestri, MD, PhD, Chief Medical Officer at Ipsen, discusses the effect of elafibranor on fatigue in patients with primary biliary cholangitis (PBC). PBC is a chronic, progressive, autoimmune, liver disease in which the bile ducts become inflamed...
