Lysosomal Disorders

The CGTLive Lysosomal Disorders specialty topic page contains video interviews with key opinion leaders discussing the most relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for Lysosomal storage diseases. It also houses the most up-to-date clinical news coverage in the field of gene and cell therapy for diseases related to lysosomal storage and dysfunction.

Latest News

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – October 4, 2024

October 4th 2024

Review top news and interview highlights from the week ending October 4, 2024.

Around the Helix: Cell and Gene Therapy Company Updates – October 2, 2024
Around the Helix: Cell and Gene Therapy Company Updates – October 2, 2024

October 2nd 2024

Myrtelle’s Canavan Disease Gene Therapy rAAV-Olig001-ASPA Decreases N-Acetylaspartate Levels in Phase 1/2 Study
Myrtelle’s Canavan Disease Gene Therapy rAAV-Olig001-ASPA Decreases N-Acetylaspartate Levels in Phase 1/2 Study

October 2nd 2024

FDA Activity Recap: September 2024 Features Acute Lymphoblastic Leukemia Trial Clearance, Multiple RMAT Designations, and More
FDA Activity Recap: September 2024 Features Acute Lymphoblastic Leukemia Trial Clearance, Multiple RMAT Designations, and More

October 1st 2024

CGTLive®’s Weekly Rewind
CGTLive®’s Weekly Rewind – September 27, 2024

September 27th 2024

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Oncology Briefings™: Coordinating the Long-Term Management of Patients With Multiple Myeloma Between Primary Care and Oncology Practices

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6th Annual Precision Medicine Symposium: An Illustrated Tumor Board

October 18-19, 2024

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Oncology Consultations®: Community and Academic Perspectives on PNH Management – How Do You Do It?

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Cases and Conversations™: Applying Guidelines to Practice for the Management of Paroxysmal Nocturnal Hemoglobinuria

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Oncology Briefings™: Selecting Therapy for Patients with Heavily Pretreated Myeloma Based on Patient and Disease-Specific Factors

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Oncology Briefings™: Case Discussions in Smoldering Myeloma—To Treat or Not to Treat?

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Medical Crossfire®: How Has the Nurse’s Role Evolved in the Management of Pediatric Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas?

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Community Practice Connections™: How Can We Optimize the Real World Application of BCMA-Targeted Therapies for Our Patients with R/R Multiple Myeloma?

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Annual Hematology Meeting: Preceding the 66th ASH Annual Meeting and Exposition

December 6, 2024

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Oncology Town Hall™: Primary Investigators Present Key Abstracts in CLL from San Diego

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Real-World Applications of Asparaginase Formulations in ALL and LBL From Pediatric/AYA to Adult Treatment: Maximizing Clinical Benefits Through Multidisciplinary Care

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Experts Address the Hottest Topics in AML: Considerations on FLT3, IDH1/2, TP53, Maintenance, Novel Combinations and Beyond!

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Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Treatment of AYA and Adult ALL

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29th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

February 27 - March 2, 2025

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Medical Crossfire®: The Experts Bridge Recent Data in Chronic Lymphocytic Leukemia With Real-World Sequencing Questions

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ACCC 50th Annual Meeting Pre-Session: Clinical Updates from San Diego on Hematologic Malignancies

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Community Practice Connections™: Pre-Conference Workshop on Immune Cell-Based Therapy

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28th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

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9th Annual Live Medical Crossfire®: Hematologic Malignancies

July 26, 2025

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