Episode 4: A Deep Dive into Rare Disease Research: Part 1
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Episode 4: A Deep Dive into Rare Disease Research: Part 1
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🎙️ 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.