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Spinal Muscular Atrophy: The Changing Definition of Success. An Expert Panel on the Evolution of SMA Care.	Rare Discussions	53:24
Growth Hormone Deficiency: Causes, Early Detection, and Treatment (Robert Rapaport, MD)	Rare Discussions	10:56
Arginine Vasopressin Deficiency (AVP-D) Overview (Christopher Romero, MD)	Rare Discussions	19:14
Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management	Rare Discussions	44:59
Prader-Willi Syndrome: Clinical Features and Early Identification	Rare Discussions	74:29
Submission of New Drug Application: Rusfertide for Polycythemia Vera	Rare Discussions	18:35
Chapter 8: Gene Therapy Discussion and Q&A	Rare Discussions	04:29
Chapter 7: Changes in Gene Therapy Programs to Lessons Learned from Recent Trials	Rare Discussions	05:18
Chapter 6: Understanding and Preparing Risk Factors Associated With AAV Gene Therapies	Rare Discussions	06:45
Chapter 5: Factors Impacting Safety and Efficacy of AAV Mediated Gene Therapies	Rare Discussions	05:54
Ch 4: Clinical Safety and Efficacy Observed in AAV Mediated Gene Therapy Programs in DMD, SMA, XLMTM	Rare Discussions	04:51
Ch 3: Mitigation Strategies to Address the Challenges in the Development of Gene Therapy Programs	Rare Discussions	05:40
Chapter 2: AAV Mediated Gene Therapies	Rare Discussions	05:07
Chapter 1: Introduction to Gene Directed Therapies	Rare Discussions	04:20
Chapter 8: Gene Therapy Discussion and Q&A	Rare Discussions	10:28
Chapter 7: Ongoing Gene Therapies in Lysosomal Disorders	Rare Discussions	08:39
Chapter 6: Gene Replacement Therapy in Lysosomal Disorders	Rare Discussions	03:38
Chapter 5: Current Treatment Landscape and Limitations	Rare Discussions	09:01
Chapter 4: Lessons Learnt from Gene Therapy Trials	Rare Discussions	02:08
Chapter 3: Immune Responses and Other Safety Concerns Related to Gene Therapies	Rare Discussions	04:50
Chapter 2: Vectors, Different Strategies, Modes of Administration, and Targets	Rare Discussions	08:59
Chapter 1: Lysosomal Disorders and the Potential for Gene Therapies	Rare Discussions	03:34
Catching the Clues, Changing the Course of Lysosomal Storage Disorders	Rare Discussions	46:37
Myasthenia Gravis Clinical Research Highlights: AAN 2025	Rare Discussions	40:47
Mastocytosis Control Test: Implications for Physicians	Rare Discussions	07:37
Fabry Disease Research Highlights	Rare Discussions	25:23
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP)	Rare Discussions	52:46
Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring	Rare Discussions	40:37
Transforming Clinical Outcomes With Early Treatment of Lysosomal Disorders	Rare Discussions	60:10
PAH Research Highlights: CHEST 2024	Rare Discussions	36:40
Consider Rare: Suspecting and Diagnosing Hereditary Angioedema (HAE)	Rare Discussions	24:37
Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion	Rare Discussions	61:37
Optimizing Therapeutic Proteins Through PEGylation: Key Parameters and Impacts	Rare Discussions	61:41
Hematologic Malignancies and Clinical Trial Participations: A Shared Decision-Making Approach	Rare Discussions	34:27
FcRn and Myasthenia Gravis	Rare Discussions	33:59
FcRn and Myasthenia Gravis: Pathophysiology	Rare Discussions	13:08
FCRn and Myasthenia Gravis: Treatment Options	Rare Discussions	20:34
Cushing’s Syndrome Treatment Research Highlights: ENDO 2024Continuing Education	Rare Discussions	33:04
CTCL: Shortening the Diagnostic Journey and Starting Treatment Early	Rare Discussions	19:51
CTCL: The Role of Dermatologists in Diagnosing and Caring for Patients	Rare Discussions	12:54

Spinal Muscular Atrophy: The Changing Definition of Success. An Expert Panel on the Evolution of SMA Care.

Rare Discussions

53:24

Growth Hormone Deficiency: Causes, Early Detection, and Treatment (Robert Rapaport, MD)

Rare Discussions

10:56

Arginine Vasopressin Deficiency (AVP-D) Overview (Christopher Romero, MD)

Rare Discussions

19:14

Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management

Rare Discussions

44:59