Rare Discussions

Breakthrough Therapy Designation to Cimzia for Antiphospholipid Syndrome	Rare Discussions	16:47
FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option	Rare Discussions	08:50
New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy	Rare Discussions	13:26
Revumenib + Chemotherapy in Newly Diagnosed AML With NPM1 Mutation: Phase 3 REVEAL-ND Study	Rare Discussions	05:13
Taletrectinib for ROS1-Positive Non-Small Cell Lung Cancer	Rare Discussions	03:30
Plasma Proteomics May Predict Disease Manifestations in ALS	Rare Discussions	10:02
Priority Review for Dersimelagon in the Treatment of EPP and XLP	Rare Discussions	14:51
A Patient’s Journey With Essential Thrombocythemia and Polycythemia Vera	Rare Discussions	16:16
FDA CDER’s Accelerating Rare Disease Cures Program	Rare Discussions	09:19
Zilganersen for the Treatment of Alexander Disease	Rare Discussions	13:47
What to Expect: 2026 MGFA Scientific Session	Rare Discussions	11:45
Topline Results From the SANRECO Trial Testing Divesiran in Polycythemia Vera	Rare Discussions	10:02
Results From the Voice of PBC Patient Survey	Rare Discussions	07:33
Approval of Imaavy (Nipocalimab) and its Impact on Patients With Warm Autoimmune Hemolytic Anemia	Rare Discussions	07:17
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

Breakthrough Therapy Designation to Cimzia for Antiphospholipid Syndrome

Rare Discussions

16:47

FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option

Rare Discussions

08:50

New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy

Rare Discussions

13:26

Revumenib + Chemotherapy in Newly Diagnosed AML With NPM1 Mutation: Phase 3 REVEAL-ND Study

Rare Discussions

05:13