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RARECast

RARECast

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

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Odcinki

621 odcinków

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