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Episodis

620 episodis

1. The Invisible Burden of a Rare Liver Disease fa 1 setmana 32:26
2. Yearning for Normal fa 2 setmanes 40:28
3. Building Better Rare Disease Data fa 3 setmanes 33:37
4. Using AI to Help Patients Get the Care They Need fa 1 mes 33:50
5. Targeting Immune Dysfunction in ALS fa 1 mes 39:47
6. When the Patient Is Left to Connect the Dots fa 1 mes 52:21
7. Enabling Patient Foundations to Build the Next Generation of Genetic Therapies fa 2 mesos 38:39
8. Using Exosomes to Tackle the Delivery Challenge of Genetic Medicines for the Brain fa 2 mesos 27:08
9. Building a Roadmap to Treat a Rare Neurodevelopmental Condition fa 2 mesos 33:55
10. The Long Road from the NICU to New Therapies fa 2 mesos 52:42
11. Stopping Guillain-Barré Syndrome at Its Source fa 3 mesos 39:13
12. From Repeated Surgery to a Precision Patch fa 3 mesos 34:56
13. Curing Sickle Cell Before Life Begins fa 3 mesos 30:56
14. Spotting Neuromuscular Red Flags fa 3 mesos 37:08
15. Expanding into a Global Rare Disease Player through Deal-Driven Innovation fa 4 mesos 51:57
16. When Geography, Cost, and Policy Become as Life-Limiting as a Disease fa 4 mesos 36:20
17. A One-Time Cell Therapy to Reset the Immune System in Autoimmune Diseases fa 4 mesos 36:49
18. Building the Infrastructure for Made‑to‑Order Gene Therapies fa 4 mesos 35:49
19. Rewiring the Rare Disease Diagnostic Odyssey fa 4 mesos 38:11
20. Emerging from a Life in the Shadows fa 5 mesos 36:36
21. Fitting Big Genes into Small Vectors fa 5 mesos 33:24
22. Targeting Iron Dysregulation in the Neurodegenerative Condition MSA fa 5 mesos 33:09
23. Long Reads, Shorter Journeys fa 5 mesos 35:23
24. The Critical Role of Patient Community-Industry Partnership in Advancing a Gene Therapy fa 6 mesos 46:20
25. Capturing the Lived Experience of Rare Disease fa 6 mesos 39:57
26. Raising Awareness of Clinical Trials fa 6 mesos 36:30
27. Using AI and Longitudinal Data to Transform Rare Disease Care fa 6 mesos 19:21
28. Cutting through the Diagnostic Maze for Rare Diseases fa 7 mesos 38:56
29. How Parents Took Development of a Gene Therapy into Their Own Hands fa 7 mesos 45:41
30. Matching the Right Therapy to the Right Child with a Rare Cancer fa 7 mesos 45:47
31. From Treating Symptoms to Addressing Causes in Rare Autoimmune Diseases fa 7 mesos 23:40
32. A Mother’s Journey to Rewrite a Neurodevelopmental Disorder fa 7 mesos 57:13
33. How a Foundation Built Its Own Drug Program for an Ultra-Rare Disease fa 8 mesos 35:50
34. From Bloodletting to Breakthroughs in PV fa 8 mesos 36:30
35. When Endpoints Miss the Point fa 8 mesos 36:47
36. Reopening the Developmental Window in Rett Syndrome with a Gene Therapy fa 8 mesos 40:52
37. A Data Strategy to Capitalize on a Multi Trillion Dollar Opportunity fa 9 mesos 26:16
38. When a Gene Echoes Through a Family fa 9 mesos 31:15
39. Changing the Autoimmune Disease Playbook with RNA-Engineered CAR T Cells fa 9 mesos 22:11
40. Rewriting Rare Disease R&D with Foundation Models fa 9 mesos 26:30
41. Resetting Aberrant Tregs Epigenetically to Treat Autoimmune Diseases fa 10 mesos 30:19
42. Finding a Ready Treatment for a Newly Discovered, Ultra-Rare Disease fa 10 mesos 32:40
43. One Rare Mother’s Quest to Rewrite Her Son’s Future with a Gene Therapy fa 10 mesos 35:46
44. Transforming TSC Epilepsy with a Precision Therapy fa 10 mesos 25:06
45. Bridging the Valley for Rare Disease Drug Development fa 1 any 41:36
46. Turning a Rare Cancer into a Call to Action fa 1 any 42:53
47. Developing a New Approach to Cystic Fibrosis to Improve Outcomes fa 1 any 29:31
48. Finding Strength in Weakness fa 1 any 25:50
49. Developing Targeted Therapies for a Rare Autoimmune Disease fa 1 any 37:41
50. A Venture Fund that Leverages Patient Experts to Target Autoimmune Diseases fa 1 any 31:01