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Submission of New Drug Application: Rusfertide for Polycythemia Vera	Rare Disease Discussions	18:35
Current Issues in Gene Therapies for Lysosomal Disorders	Rare Disease Discussions	106:41
Theranostics and Lysosomal Disorders	Rare Disease Discussions	106:09
Expanded Applications of AI in Lysosomal Disorders	Rare Disease Discussions	121:27
Organoids and Lab-Grown Models in Lysosomal Disorders	Rare Disease Discussions	96:02
Nanotechnology and Lysosomal Disorders	Rare Disease Discussions	50:18
AI in Medicine: Transforming the Landscape of Tissue-Based Diagnostics	Rare Disease Discussions	52:29
Chapter 8: Gene Therapy Discussion and Q&A	Rare Disease Discussions	04:29
Chapter 7: Changes in Gene Therapy Programs to Lessons Learned from Recent Trials	Rare Disease Discussions	05:18
Chapter 6: Understanding and Preparing Risk Factors Associated With AAV Gene Therapies	Rare Disease Discussions	06:45
Chapter 5: Factors Impacting Safety and Efficacy of AAV Mediated Gene Therapies	Rare Disease Discussions	05:54
Ch 4: Clinical Safety and Efficacy Observed in AAV Mediated Gene Therapy Programs in DMD, SMA, XLMTM	Rare Disease Discussions	04:51
Ch 3: Mitigation Strategies to Address the Challenges in the Development of Gene Therapy Programs	Rare Disease Discussions	05:40
Chapter 2: AAV Mediated Gene Therapies	Rare Disease Discussions	05:07
Chapter 1: Introduction to Gene Directed Therapies	Rare Disease Discussions	04:20
Chapter 8: Gene Therapy Discussion and Q&A	Rare Disease Discussions	10:28
Chapter 7: Ongoing Gene Therapies in Lysosomal Disorders	Rare Disease Discussions	08:39
Chapter 6: Gene Replacement Therapy in Lysosomal Disorders	Rare Disease Discussions	03:38
Chapter 5: Current Treatment Landscape and Limitations	Rare Disease Discussions	09:01
Chapter 4: Lessons Learnt from Gene Therapy Trials	Rare Disease Discussions	02:08
Chapter 3: Immune Responses and Other Safety Concerns Related to Gene Therapies	Rare Disease Discussions	04:50
Chapter 2: Vectors, Different Strategies, Modes of Administration, and Targets	Rare Disease Discussions	08:59
Chapter 1: Lysosomal Disorders and the Potential for Gene Therapies	Rare Disease Discussions	03:34
Catching the Clues, Changing the Course of Lysosomal Storage Disorders	Rare Disease Discussions	46:37
Consider Rare: Suspecting and Diagnosing CIDP	Rare Disease Discussions	28:45
Lysosomal Disorders and the Brain	Rare Disease Discussions	58:01
Case Studies in Diagnosing and Managing FOP	Rare Disease Discussions	50:57
Immune Thrombocytopenia (ITP) Research Highlights: ISTH 2025	Rare Disease Discussions	27:11
Immune Thrombotic Thrombocytopenic Purpura (iTTP) Research Highlights: ISTH 2025	Rare Disease Discussions	25:17
Hemophilia Research Highlights: ISTH 2025	Rare Disease Discussions	44:48
Myasthenia Gravis Clinical Research Highlights: AAN 2025	Rare Disease Discussions	40:47
Mastocytosis Control Test: Implications for Physicians	Rare Disease Discussions	07:37
Skeletal Involvement in Lysosomal Disorders	Rare Disease Discussions	56:43
Fabry Disease Research Highlights	Rare Disease Discussions	25:23
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP)	Rare Disease Discussions	52:46
Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring	Rare Disease Discussions	40:37
Transforming Clinical Outcomes With Early Treatment of Lysosomal Disorders	Rare Disease Discussions	60:10
PAH Research Highlights: CHEST 2024	Rare Disease Discussions	36:40
Consider Rare: Suspecting and Diagnosing Hereditary Angioedema (HAE)	Rare Disease Discussions	24:37
Improving Health Equity in Hereditary Angioedema (HAE): A Panel Discussion	Rare Disease Discussions	61:37

Submission of New Drug Application: Rusfertide for Polycythemia Vera

Rare Disease Discussions

18:35

Current Issues in Gene Therapies for Lysosomal Disorders

Rare Disease Discussions

106:41

Theranostics and Lysosomal Disorders

Rare Disease Discussions

106:09

Expanded Applications of AI in Lysosomal Disorders

Rare Disease Discussions

121:27