by Madaline Spencer | Mar 7, 2025
Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses a recent study examining the effectiveness of idursulfase in young patients with mucopolysaccharidosis type II (MPS II). MPS II is a rare...
by Madaline Spencer | Mar 6, 2025
Karen Bean, Health Economist at Orchard Therapeutics, discusses a recent study comparing the effect of gene therapy in treated versus untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD). MLD is a rare genetic condition...
by Madaline Spencer | Mar 5, 2025
Daniel Lewi, Founder of the CATS Foundation, and Kathleen Flynn, CEO of the National Tay-Sachs & Allied Diseases Association (NTSAD), discuss an industry-advocacy collaboration to optimize clinical trial design through a patient-centric approach. An...
by Madaline Spencer | Feb 28, 2025
February 28 Is Rare Disease Day! Rare Disease Day, observed on the last day of February every year, is a reminder of the challenges faced by those living with a rare disease. Established in 2008 by EURORDIS (The European Organisation for Rare Diseases), the day serves...
by Madaline Spencer | Feb 27, 2025
Alix Arnaud, Director of Health Economic and Value Assessment BP at Sanofi, discusses the effects of caplacizumab combination therapy in patients with acquired thrombotic thrombocytopenic purpura (TTP). TTP is a rare blood disorder characterized by low...
