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...
by Madaline Spencer | Feb 26, 2025
Aneal Khan, MD, President of MAGIC Clinic (Metabolics and Genetics in Canada), discusses five year end-of-study results from the FACTs trial in patients in Fabry disease. Fabry disease is a rare lysosomal storage disease characterized by a deficiency of...
