Note sull'episodio
đď¸ Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin
đ¤ Guest: Mathias Schmidt
đ Release Date: 7/11/2025
đŹ Topic: Breaking the Blood-Brain Barrier, Compassionate Use, and the Power of Patient-Centered Innovation
Episode Overview
In this episode of These Kids Canât Wait, weâre joined by Mathias Schmidt, President and CEO of Armagen and Executive Fellow at JCR Pharmaceuticals. With over 20 years in rare disease research, Dr. Schmidt discusses how science and empathy intersect in the development of treatments for lysosomal storage diseasesâparticularly those affecting the brain. From pioneering enzyme therapies that cross the blood-brain barrier to founding The Lost Enzyme Project, Mathias offers a hope ...Â
Parole chiave
Beta-mannosidosisThe Lost Enzyme Project