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Rare Discussions

CheckRare Editors

Conversations with the leaders advancing rare disease care. Rare Discussions is CheckRare's flagship interview podcast featuring conversations with leading physicians, researchers, patient advocates, and industry experts. These episodes explore advances in diagnosis, treatment, research, and patient care across the rare disease landscape. Whether highlighting groundbreaking therapies or sharing expert clinical perspectives, Rare Discussions highlights the experts who are shaping the future of rare disease medicine.

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Эпизоды

210 эпизодов

  1. The Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease 11:58
  2. UCB Study Highlights the Burden of Ocular Myasthenia Gravis 11:15
  3. Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF 9:14
  4. Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis 7:42
  5. Garetosmab Demonstrates Sustained Efficacy in Adults With Fibrodysplasia Ossificans Progressiva 5:10
  6. C3G and the Pediatric VALIANT Study 10:49
  7. A Family’s Journey with Prader-Willi Syndrome 10:22
  8. Molecular Changes Associated With Teprotumumab Treatment in Thyroid Eye Disease 6:35
  9. Mirum's Livmarli Achieves Primary Endpoint in Phase 3 EXPAND Study 18:45
  10. How Bispecific Antibodies Are Changing Multiple Myeloma Treatment 12:05
  11. Miv-cel Shows Durable Results in Stiff Person Syndrome and Myasthenia Gravis 15:53
  12. Z-Basivarsen Shows Functional Improvements in One-Year Data for Myotonic Dystrophy Type 1 9:02
  13. FDA Approves Gazyva to Reduce Relapse Risk in Patients With Idiopathic Nephrotic Syndrome 10:37
  14. Efficacy of Vosoritide to Treat Hypochondroplasia 12:24
  15. Efficacy of Infigratinib on Achondroplasia 15:59
  16. Breakthrough Therapy Designation to Cimzia for Antiphospholipid Syndrome 16:47
  17. FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option 8:50
  18. New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy 13:26
  19. Revumenib + Chemotherapy in Newly Diagnosed AML With NPM1 Mutation: Phase 3 REVEAL-ND Study 5:13
  20. Taletrectinib for ROS1-Positive Non-Small Cell Lung Cancer 3:30
  21. Plasma Proteomics May Predict Disease Manifestations in ALS 10:02
  22. Priority Review for Dersimelagon in the Treatment of EPP and XLP 14:51
  23. A Patient’s Journey With Essential Thrombocythemia and Polycythemia Vera 16:16
  24. FDA CDER’s Accelerating Rare Disease Cures Program 9:19
  25. Zilganersen for the Treatment of Alexander Disease 13:47
  26. What to Expect: 2026 MGFA Scientific Session 11:45
  27. Topline Results From the SANRECO Trial Testing Divesiran in Polycythemia Vera 10:02
  28. Results From the Voice of PBC Patient Survey 7:33
  29. Approval of Imaavy (Nipocalimab) and its Impact on Patients With Warm Autoimmune Hemolytic Anemia 7:17
  30. Spinal Muscular Atrophy: The Changing Definition of Success. An Expert Panel on the Evolution of SMA Care. 53:24
  31. Growth Hormone Deficiency: Causes, Early Detection, and Treatment (Robert Rapaport, MD) 10:56
  32. Arginine Vasopressin Deficiency (AVP-D) Overview (Christopher Romero, MD) 19:14
  33. Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management 44:59
  34. Prader-Willi Syndrome: Clinical Features and Early Identification 1:14:29
  35. Submission of New Drug Application: Rusfertide for Polycythemia Vera 18:35
  36. Chapter 8: Gene Therapy Discussion and Q&A 4:29
  37. Chapter 7: Changes in Gene Therapy Programs to Lessons Learned from Recent Trials 5:18
  38. Chapter 6: Understanding and Preparing Risk Factors Associated With AAV Gene Therapies 6:45
  39. Chapter 5: Factors Impacting Safety and Efficacy of AAV Mediated Gene Therapies 5:54
  40. Ch 4: Clinical Safety and Efficacy Observed in AAV Mediated Gene Therapy Programs in DMD, SMA, XLMTM 4:51
  41. Ch 3: Mitigation Strategies to Address the Challenges in the Development of Gene Therapy Programs 5:40
  42. Chapter 2: AAV Mediated Gene Therapies 5:07
  43. Chapter 1: Introduction to Gene Directed Therapies 4:20
  44. Chapter 8: Gene Therapy Discussion and Q&A 10:28
  45. Chapter 7: Ongoing Gene Therapies in Lysosomal Disorders 8:39
  46. Chapter 6: Gene Replacement Therapy in Lysosomal Disorders 3:38
  47. Chapter 5: Current Treatment Landscape and Limitations 9:01
  48. Chapter 4: Lessons Learnt from Gene Therapy Trials 2:08
  49. Chapter 3: Immune Responses and Other Safety Concerns Related to Gene Therapies 4:50
  50. Chapter 2: Vectors, Different Strategies, Modes of Administration, and Targets 8:59