Note sull'episodio

🎙️ Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin

🎤 Guest: Mathias Schmidt

📅 Release Date: 7/11/2025

🔬 Topic: Breaking the Blood-Brain Barrier, Compassionate Use, and the Power of Patient-Centered Innovation

Episode Overview

In this episode of These Kids Can’t Wait, we’re 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 diseases—particularly those affecting the brain. From pioneering enzyme therapies that cross the blood-brain barrier to founding The Lost Enzyme Project, Mathias offers a hope ... 

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Parole chiave
Beta-mannosidosisThe Lost Enzyme Project