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Episode 8: Turning Hope into Action

Episode 8: Turning Hope into Action

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About this listen

🎙️ 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 hopeful, grounded vision for what’s possible when patients are at the center of innovation.

Key Takeaways

✔️ How JCR’s J-Brain Cargo technology delivers enzymes past the blood-brain barrier

✔️ Why focusing on quality of life—rather than a cure—is a powerful shift in mindset

✔️ Reflections on the emotional and ethical dimensions of working in rare disease

Resources & Links

🌐 Learn more: thelostenzymeproject.org

📢 Follow us on social media:

https://www.linkedin.com/company/the-lost-enzyme-project

https://www.instagram.com/thelostenzymeproject/

https://www.facebook.com/people/The-Lost-Enzyme-Project/61551541563692/

📩 Contact us:

Email us at admin@thelostenzymeproject.org

Producer: alex@thelostenzymeproject.org

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Stay informed by subscribing to These Kids Can’t Wait on Spotify, Apple Podcasts, Youtube or Amazon music. If you find this episode valuable, please leave a review to help raise awareness for rare disease research.

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