Lysosomal Disorders

Latest News

January 8th 2025
Around the Helix: Cell and Gene Therapy Company Updates – January 8, 2025

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Heather Lau, MD, MS
January 7th 2025

Ultragenyx Puts BLA for MPSIII Gene Therapy UX111 in Front of FDA

January 6th 2025

Arbor Biotechnologies’ CRISPR-Based Gene Editing Therapy ABO-101 Cleared for US Trial in Primary Hyperoxaluria Type 1

CGTLive®’s Weekly Rewind
January 3rd 2025

CGTLive®’s Weekly Rewind – January 3, 2025

January 2nd 2025

CGTLive's 2024 Pillars of Progress: Top News in Lysosomal Disorders

Latest Videos

CME Content

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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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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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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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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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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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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

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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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Immune Therapies in Practice in 2026: Cellular Therapies, ADCs, Bispecifics and Beyond
In-Person Event

Immune Therapies in Practice in 2026: Cellular Therapies, ADCs, Bispecifics and Beyond

May 22, 2026

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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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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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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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