🎙️ 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.

🎙️ Host: Erin Hubbard

🎤 Guest: Sarah Forsman

📅 Release Date: 2/28/2025

🔬 Topic: Life with Alpha-Mannosidosis and the Impact of Early Treatment

Episode Overview

In this episode of These Kids Can’t Wait, host Erin Hubbard speaks with Sarah Forsman, a patient living with Alpha-Mannosidosis. Sarah shares her personal journey, including undergoing a bone marrow transplant at the age of four, and how this early intervention has shaped her life.

From navigating the challenges of a rare disease to advocating for greater awareness, Sarah offers a unique and inspiring perspective on what it means to live with Alpha-Mannosidosis. This conversation sheds light on the realities of managing a lysosomal storage disorder, the importance of early diagnosis and treatment, and the resilience of the rare disease community.

Key Takeaways

✔️ Sarah’s experience with Alpha-Mannosidosis and her early treatment journey

✔️ The impact of a bone marrow transplant on disease progression

✔️ Challenges and triumphs of living with a rare disease

✔️ The importance of research, advocacy, and patient support networks

Resources & Links

🌐 Learn more about The Lost Enzyme Project: https://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 to get in contact with our producer at alex@thelostenzymeproject.org

Sarah’s Blog: https://achievetheimpossibletoday.com/blog/

ISMRD: https://www.ismrd.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: Lorena Lomelin and Phoebe Wang

🎤 Guest: Dr. Virginia Kimonis

📅 Release Date:2/28/2025

🔬 Topic: A deep dive into rare disease research.

Episode Summary:

In this episode of These Kids Can’t Wait, hosts Lorena Lomelin and Phoebe Wang sits down with Dr. Virginia Kimonis, a nationally renowned expert in genetics and clinical biochemical genetics, to discuss her groundbreaking work on Beta-Mannosidosis and the development of a novel treatment. Dr. Kimonis shares her journey into rare disease research, the challenges of treating lysosomal storage disorders, and the innovative science behind The Lost Enzyme Project. We also explore the impact of this research on patients and families, the funding challenges in rare disease research, and the future of treatments for ultra-rare conditions.

Key Takeaways:

✔️ What lysosomal storage disorders are and why they’re challenging to treat

✔️ An overview of Beta-Mannosidosis, its symptoms and the challenges faced

✔️ The process of developing a treatment, from preclinical research to clinical trials

✔️ The future of rare disease research and the potential for gene therapy

Resources & Links

🌐 Learn more: https://thelostenzymeproject.org/ or https://bit.ly/lostenzyme

📢 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 to get in contact with our producer at alex@thelostenzymeproject.org

Dr. Kimonis’s lab: https://www.mammag.uci.edu/kimonis/index.asp

Subscribe & Stay Connected

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

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

📅 Release Date:2/28/2025

🔬 Topic: Story Behind the Lost Enzyme Project

Episode Overview

In this inaugural episode of These Kids Can’t Wait, we are joined by the co-founders of The Lost Enzyme Project—Erin Hubbard, Laurel Gregier, and Lorena Lomelin. As parents of children affected by ultra-rare lysosomal storage disorders, they share their deeply personal experiences, the challenges of advocating for research in rare diseases, and the motivation behind launching this initiative.

This discussion highlights the urgent need for scientific progress, the barriers to treatment development, and the power of patient-led advocacy in accelerating change.

Key Takeaways

✔️ The personal journeys that led to the creation of The Lost Enzyme Project

✔️ The organization’s mission to drive research and innovation for rare diseases

✔️ Challenges in the rare disease space, including funding gaps and limited awareness

✔️ The importance of collaboration between families, researchers, and biotech leaders

Resources & Links

🌐 Learn more: https://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 to get in contact with our producer at 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.