🎙️ 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

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📩 Contact us:

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