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Episode 8: Turning Hope into Action
šļø Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin
š¤ Guest: Mathias Schmidt
š Release Date: 7/11/2025
š¬ Topic: Breaking the Blood-Brain Barrier, Compassionate Use, and the Power of Patient-Centered Innovation
Episode Overview
In this episode of These Kids Canāt Wait, weāre 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 diseasesāparticularly 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 whatās possible when patients are at the center of innovation.
Key Takeaways
āļø How JCRās J-Brain Cargo technology delivers enzymes past the blood-brain barrier
āļø Why focusing on quality of lifeārather than a cureāis a powerful shift in mindset
āļø Reflections on the emotional and ethical dimensions of working in rare disease
Resources & Links
š Learn more: thelostenzymeproject.org
š¢ 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/
š© Contact us:
Email us at [email protected]
Producer: [email protected]
Subscribe & Stay Connected
Stay informed by subscribing to These Kids Canāt 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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By Erin Hubbardšļø Hosts: Erin Hubbard, Laurel Gregier, and Lorena Lomelin
š¤ Guest: Mathias Schmidt
š Release Date: 7/11/2025
š¬ Topic: Breaking the Blood-Brain Barrier, Compassionate Use, and the Power of Patient-Centered Innovation
Episode Overview
In this episode of These Kids Canāt Wait, weāre 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 diseasesāparticularly 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 whatās possible when patients are at the center of innovation.
Key Takeaways
āļø How JCRās J-Brain Cargo technology delivers enzymes past the blood-brain barrier
āļø Why focusing on quality of lifeārather than a cureāis a powerful shift in mindset
āļø Reflections on the emotional and ethical dimensions of working in rare disease
Resources & Links
š Learn more: thelostenzymeproject.org
š¢ 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/
š© Contact us:
Email us at [email protected]
Producer: [email protected]
Subscribe & Stay Connected
Stay informed by subscribing to These Kids Canāt 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.