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.
September 6th 2024
Review top news and interview highlights from the week ending September 6, 2024.
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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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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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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Oncology Briefings™: Case Discussions in Smoldering Myeloma—To Treat or Not to Treat?
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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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Experts Address the Hottest Topics in AML: Considerations on FLT3, IDH1/2, TP53, Maintenance, Novel Combinations and Beyond!
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Putting Your Stamp on the Next Generation of Care in CML — Improving Outcomes from Frontline to R/R Treatment Settings
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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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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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Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Treatment of AYA and Adult ALL
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How Do We Treat GVHD Today…And Tomorrow? Treatment Algorithms for Key Decision Points in Prophylaxis Care, Acute, and Chronic Disease
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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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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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28th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma
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Community Practice Connections™: Pre-Conference Workshop on Immune Cell-Based Therapy
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9th Annual Live Medical Crossfire®: Hematologic Malignancies
July 26, 2025
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Gene Therapy for CLN6 Batten Disease Shows Positive Interim Results
August 3rd 2019Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.