🎙️ Hosts: Erin Hubbard and Laurel Gregier

🎤 Guest: Dr. Paul Orchard

📅 Release Date: 5/12/2025

🔬 Topic: Bone Marrow Transplants, Gene Therapy, and the Future of Rare Disease Treatment

Episode Overview

In this episode of These Kids Can’t Wait, we are joined by Dr. Paul Orchard, Medical Director of the Inherited Metabolic and Storage Disease Transplantation Program at the University of Minnesota. Dr. Orchard brings decades of experience treating ultra-rare lysosomal disorders through stem cell transplantation and emerging gene therapies. Together with hosts Erin and Laurel, he walks us through the science, challenges, and hope behind bone marrow transplants for rare diseases, sharing insights from his career, reflections on patient care, and the future of life-saving treatment.

Key Takeaways

✔️ How bone marrow transplantation works for lysosomal storage disorders

✔️ The difference between cord blood and unrelated donor stem cells

✔️ The impact of early diagnosis and newborn screening

✔️ Limitations of enzyme replacement therapy and the promise of gene therapy

✔️ How families and advocates can help shape research and access to treatment

✔️ The emotional and medical realities of caring for ultra-rare patients

Resources & Links

🌐 Learn more: thelostenzymeproject.org

📢 Follow us on social media:

https://www.linkedin.com/feed/

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

Subscribe & Stay Connected

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.

Episode 5: A Deep Dive into Rare Disease Research: Part 2

🎙️ Hosts: Erin Hubbard, Laurel Gregier

🎤 Guest: Dr. Martin

📅 Release Date: 4/21/2025

🔬 Topic: Advancing Research for Beta-Mannosidosis

Episode Overview

In Part 2 of our conversation with Dr. Angela Martin, we are continuing our conversation on rare disease research. Building on our earlier discussion of enzyme replacement therapy and the challenges of treating beta-mannosidosis, this episode explores how awareness, advocacy, and day-to-day lab work come together to drive progress.

Dr. Martin shares her passion for science, the emotional impact of working closely with families, and how conferences like WORLD play a pivotal role in forging critical partnerships. We also talk about the real-life stories and responsibilities behind the science, and what keeps researchers like Dr. Martin going in the face of uncertainty.

Key Takeaways

✔️ The importance of awareness and early diagnosis in rare disease treatment

✔️ How parent-led advocacy accelerates scientific progress

✔️ The daily grind—and joy—of conducting rare disease research

✔️ The pivotal role of conferences like WORLD in connecting research teams with potential biotech partners

✔️ How families and supporters can meaningfully contribute to ongoing research

Resources & Links

🌐 Learn more: thelostenzymeproject.org

📢 Follow us on social media:

https://www.linkedin.com/feed/

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

Subscribe & Stay Connected

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.

🎙️ Hosts: Erin Hubbard and Laurel Gregier

🎤 Guest: Dr. Angela Martin

📅 Release Date: 4/8/2025

🔬 Topic: Understanding Beta-Mannosidosis and the Innovation Behind Enzyme Replacement Therapy

Episode Overview

In this episode of These Kids Can’t Wait, hosts Erin Hubbard and Laurel Gregier are joined by Dr. Angela Martin, a postdoctoral researcher at the Kimonis Laboratory and a vital force in The Lost Enzyme Project. Together, we discuss beta-mannosidosis, a rare lysosomal storage disorder, and the innovative efforts underway to develop treatments, including a promising new therapy using J-Brain Cargo technology.

Key Takeaways

✔️ What beta-mannosidosis is and why it's so difficult to diagnose and treat

✔️ How J-Brain Cargo technology enables enzyme delivery across the blood-brain barrier

✔️ The stages of drug development—from lab to clinical trials

✔️ Why underdiagnosis is a major issue for ultra-rare diseases

✔️ The power of international collaboration in rare disease innovation

Resources & Links

🌐 Learn more: thelostenzymeproject.org

📢 Follow us on social media:

https://www.linkedin.com/feed/

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

Subscribe & Stay Connected

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.