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Folgen

621 Folgen

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