Lysosomal Disorders

Latest News

January 1st 2024
CGTLive's Clinical Trials to Watch in 2024

These, among many others, are some of the key clinical trials of gene, cell, and regenerative therapies that the CGTLive staff will be following throughout 2024.

December 31st 2023

CGTLive's Pillars of Progress 2023: Top FDA Approval News in Cell and Gene Therapy

December 27th 2023

Around the Helix: Cell and Gene Therapy Company Updates – December 27, 2023

December 26th 2023

CGTLive's Pillars of Progress 2023: Top News in Lysosomal Disorders

December 20th 2023

Around the Helix: Cell and Gene Therapy Company Updates – December 20, 2023

Latest Videos

CME Content

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Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice
Video

Addressing Racial Disparities in Multiple Myeloma: Forging a Path Towards Health Equity in Clinical Trials and Everyday Practice

Craig Emmitt Cole, MD; Sikander Ailawadhi, MD; Irene Ghobrial, MD; Monique Hartley-Brown, MD, MMSc

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Medical Crossfire® Navigating the Chronic Myeloid Leukemia Landscape: Treatment Strategies, Mechanisms, and Patient-Centered Care
Virtual Event

Medical Crossfire® Navigating the Chronic Myeloid Leukemia Landscape: Treatment Strategies, Mechanisms, and Patient-Centered Care

December 15, 2025

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Tackling Oncologic Emergencies in Patients Treated with High-Dose Methotrexate
Virtual Event

Tackling Oncologic Emergencies in Patients Treated with High-Dose Methotrexate

December 17, 2025

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Oncology Town Hall: Shaping the Future of ALL Treatment Through Innovation in T-Cell Therapies
Virtual Event

Oncology Town Hall: Shaping the Future of ALL Treatment Through Innovation in T-Cell Therapies

December 18, 2025

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Evolving Strategies and Emerging Agents in PTCL: Subtypes, Mutations, and Modern Therapies
Virtual Event

Evolving Strategies and Emerging Agents in PTCL: Subtypes, Mutations, and Modern Therapies

December 19, 2025

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Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment
Multimedia

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

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Cases and Conversations™: Optimizing Patient Outcomes in Pediatric/AYA and Adult ALL/LBL With Asparaginase Therapies and Combinations
Video

Cases and Conversations™: Optimizing Patient Outcomes in Pediatric/AYA and Adult ALL/LBL With Asparaginase Therapies and Combinations

Daniel J. DeAngelo, MD, PhD; Shira Dinner, MD; Stephen P. Hunger, MD; Jae H. Park, MD

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Advances in ALL Management Across the Patient Spectrum: Integrating Innovation and Precision Care
Video

Advances in ALL Management Across the Patient Spectrum: Integrating Innovation and Precision Care

Elias Jabbour, MD; Hagop Kantarjian, MD; Aaron Logan, MD, PhD, MPhil; Rob Pieters, MD, PhD; Claire Roddie, MD, PhD

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PER® Tumor Board: Charting a New Course in the Treatment of Newly Diagnosed Multiple Myeloma
Video

PER® Tumor Board: Charting a New Course in the Treatment of Newly Diagnosed Multiple Myeloma

Ajai Chari, MD; Maria-Victoria Mateos, MD, PhD; Mohamad Mohty, MD, PhD

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Cases and Conversations: Real-World Insights in Advancing Management of Myelofibrosis and Polycythemia Vera – How the Experts Apply the Latest Developments in Care
Video

Cases and Conversations: Real-World Insights in Advancing Management of Myelofibrosis and Polycythemia Vera – How the Experts Apply the Latest Developments in Care

Naveen Pemmaraju, MD; Shannon Elf, PhD; Angela Fleischman, MD, PhD; Bethan Psaila, MD

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Medical Crossfire®: Navigating Chronic GVHD Prophylaxis and Treatment – Targeted Strategies to Elevate Patient Outcomes
Video

Medical Crossfire®: Navigating Chronic GVHD Prophylaxis and Treatment – Targeted Strategies to Elevate Patient Outcomes

Corey Cutler, MD, MPH, FRCP(C); Amin M. Alousi, MD; Mehdi Hamadani, MD; Anna Sureda, MD, PhD

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Medical Crossfire®: Experts Debate the Hottest Topics in AML – FLT3, IDH1/2, KMT2Ar, NPM1, Secondary AML, Maintenance, Novel Combinations, and Emerging Therapies
Video

Medical Crossfire®: Experts Debate the Hottest Topics in AML – FLT3, IDH1/2, KMT2Ar, NPM1, Secondary AML, Maintenance, Novel Combinations, and Emerging Therapies

Naval Daver, MD; Daniel A. Pollyea, MD; Gail J. Roboz, MD; Amer Zeidan, MBBS, MHS

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How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma
Video

How We Do It®: New Combinations Reshaping Treatment Decision-Making in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Cristina Gasparetto, MD; Katja Weisel, MD

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Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET
Video

Medical Crossfire®: Bridging Expert Guidance and Evidence-Based Recommendations in the Ever-Evolving Treatment of MF, PV, and ET

Rami S. Komrokji, MD; Ghaith Abu-Zeinah, MD; Gabriela Hobbs, MD; Barbara Mora, MD

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From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice
Video

From Barriers to Breakthroughs in Multiple Myeloma: How Trials Compare to the Real-World Management of Patients in Your Practice

Krina Patel, MD, MSc; Alfred L. Garfall, MD; Ajay K. Nooka, MD, MPH, FACP

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Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?
Video

Show Me the Data: How Do We Navigate the Latest Evidence on Novel Therapies and Trials Across GvHD Prophylaxis, Acute, and Chronic Care?

Corey Cutler, MD, MPH, FRCPC; Amin M. Alousi, MD; Yi-Bin Chen, MD; Carrie Lynn Kitko, MD

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Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios
Video

Medical Crossfire®: New Horizons in Acute Lymphoblastic Leukemia—How The Experts Apply New Evidence from AYA to Adult Scenarios

Elias Jabbour, MD; Hagop M. Kantarjian, MD; Aaron Logan, MD, PhD, MPhil; Rob Pieters, MD, PhD; Claire Roddie, MD, PhD

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Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead
Video

Show Me the Data™: Pushing CAR Ts Forward Across Lymphoid Malignancies…Accelerating Access in the Community and the Treatment Strategies for the Road Ahead

Alexey V. Danilov, MD, PhD; Leidy L. Isenalumhe, MD, MS; Lori A. Leslie, MD; Mazyar Shadman, MD, MPH; Jason R. Westin, MD, MS, FACP

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Navigating the Next Wave in BTK Inhibition Strategies: Maximizing Clinical Benefit in CLL and MCL, What’s Next in B-Cell Malignancies
Video

Navigating the Next Wave in BTK Inhibition Strategies: Maximizing Clinical Benefit in CLL and MCL, What’s Next in B-Cell Malignancies

Catherine C. Coombs, MD, MS; Paolo Ghia, MD, PhD; Tycel J. Phillips, MD; Jeffrey V. Matous, MD; Grzegorz S. Nowakowski, MD, FASCO

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SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients
Case-based Simulation

SimulatEd™ in Multiple Myeloma: Personalizing Care Plans to Treat Newly Diagnosed Patients

Amrita Krishnan, MD

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30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma
In-Person Event

30th Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas, and Myeloma

February 26-28, 2026

Register now!
Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes
Video

Medical Crossfire®: Expert Interpretations of the Latest Data in CLL Management – Understanding the Impact of Optimal Treatment Selection on Patient Outcomes

Jennifer R. Brown, MD, PhD; Alexey V. Danilov, MD, PhD; Matthew S. Davids, MD, MMSc

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Upstate New York Hematology & Hematologic Oncology Conference: A Review of the 67th ASH Annual Meeting
In-Person + Virtual Event

Upstate New York Hematology & Hematologic Oncology Conference: A Review of the 67th ASH Annual Meeting

April 25-26, 2026

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

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

André H. Goy, MD; Jorge E. Cortes, MD; Sagar Lonial, MD, FACP

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Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer
Multimedia

Community Practice Connections™: Tailored Treatment Approaches for Older Patients With Advanced HR+/HER2– Breast Cancer

Hope S. Rugo, MD, FASCO; Neil M. Iyengar, MD; Komal Jhaveri, MD, FACP

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Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma
Video

Medical Crossfire®: Harnessing the Power of Modern Therapies in Newly Diagnosed Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

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Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights
Video

Cases & Conversations™: Transforming AML Care—Precision Strategies, Evolving Therapies, and Clinical Insights

Gail J. Roboz, MD; Hagop Kantarjian, MD; Catherine Lai, MD, MPH; Amer Zeidan, MBBS, MHS

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Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma
Video

Cases and Conversations™: Sorting Through the Expanding Treatment Options for Patients with Relapsed/Refractory Multiple Myeloma

C. Ola Landgren, MD, PhD; Mohamad Mohty, MD, PhD; Surbhi Sidana, MD

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Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches
Video

Medical Crossfire®: Improving Patient Outcomes in Myeloproliferative Neoplasms With Novel Therapeutic Approaches

Rami Komrokji, MD; Gabriela Hobbs, MD; John Mascarenhas, MD; Naveen Pemmaraju, MD

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10th Annual Live Medical Crossfire®: Hematologic Malignancies
In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

Register now!
Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma
Video

Expert Perspectives on Applying Recent Evidence to Practice in the Management of Newly Diagnosed Multiple Myeloma

Amrita Krishnan, MD; Meletios Athanasios (Thanos) C. Dimopoulos, MD; Shaji Kumar, MD; Katja Weisel, MD

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Imaging Matters: Decoding PSMA PET for Better Decision-Making
Multimedia

Imaging Matters: Decoding PSMA PET for Better Decision-Making

Jeremie Calais, MD, PhD; Tanya B. Dorff, MD; Scott T. Tagawa, MD, MS, FACP, FASCO

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SimulatEd™: Partnering for Precision – A Framework for Personalized Care Planning in Acute Lymphoblastic Leukemia
Case-based Simulation

SimulatEd™: Partnering for Precision – A Framework for Personalized Care Planning in Acute Lymphoblastic Leukemia

Jae Park, MD

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9th Annual School of Nursing Oncology™
Video

9th Annual School of Nursing Oncology™

Beth Faiman, PhD, MSN, APN-BC, BMTCN, AOCN, FAAN, FAPO; Beth Sandy, MSN, CRNP, FAPO; Lindsay Adkins, MSN, FNP-BC, BMTCN; Jeneth Aquino, DNP, FNP-BC; Casey Gormley, MSN, FNP-C, AOCNP; Heather J. Jackson, PhD, FNP-BC; Kelsey Martin, AG-ACNP-BC, AOCNP; Nerina T. McDonald, PA-C; Lauren Verity Moore, DNP, MSN, AGACNP-BC; Faith A. Mutale, DNP, CRNP; Tiffany Richards, PhD, ANP-BC, AOCNP; Emily Skotte, DNP, MSN, ACNP-BC; Leslie Smith, DNP, RN, APRN-CNS, AOCNS, BMTCN; Saneese Stephen, PA-C, MPAS; Sara M. Tinsley-Vance, PhD, APRN, AOCN

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Cases and Conversations™: Optimizing Outcomes in PV and ET—Evolving Standards, Symptom Management, and Therapeutic Innovation
Video

Cases and Conversations™: Optimizing Outcomes in PV and ET—Evolving Standards, Symptom Management, and Therapeutic Innovation

John O. Mascarenhas, MD; Marina Kremyanskaya, MD, PhD; Douglas A. Tremblay, MD

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