Lysosomal Disorders

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David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases

Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth

Sowmya Viswanathan, PhD, on Translating Cell Therapies to the Clinic at ISCT 2024

Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility

Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary

Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology

Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy

Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials

Maria Escolar, MD, the chief medical officer of Forge Biologics

Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease

John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies

John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1

CME Content

Video

(COPE) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

Video

(CME) Optimizing Management of Ocular Toxicity in Cancer Patients: The Role of Ophthalmologists in the Spectrum of Care

Asim V. Farooq, MD; Joann Kang, MD; Nita Lee, MD, MPH

Multimedia

Community Practice Connections™: 6th Annual Precision Medicine Symposium – An Illustrated Tumor Board

Andre H. Goy, MD; Monique Hartley-Brown, MD, MMSc (Guest Co-Chair); Chiara Battelli, MD, PhD; Marios Giannakis, MD, PhD; Shail Maingi, MD; Aparna Parikh, MD

Video

Addressing Pivotal Updates in AML – Expert Perspectives on Individualizing Management for Special Populations

Eytan M. Stein, MD; Marina Kremyanskaya, MD, PhD; Hannah Levavi, MD

Video

Innovations in MDS Care: Navigating New Treatment Paradigms, Breakthrough Therapies, and Emerging Clinical Evidence

Rami Komrokji, MD; Maen Hussein, MD

In-Person + Virtual Event

Annual Hematology Meeting: Preceding the 67th ASH Annual Meeting and Exposition

December 5, 2025

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

Virtual Event

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

December 19, 2025

Multimedia

Rounds with Dr Jabbour: Breaking Barriers in ALL Treatment

Elias Jabbour, MD

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

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

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

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

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

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

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

Case-based Simulation

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

Amrita Krishnan, MD

In-Person Event

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

February 26-28, 2026

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

In-Person + Virtual Event

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

April 25-26, 2026

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

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

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

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

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

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

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

In-Person Event

10th Annual Live Medical Crossfire®: Hematologic Malignancies

July 11, 2026

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

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

More News

Around the Helix: Cell and Gene Therapy Company Updates – August 6, 2025

ByNoah Stansfield

August 6th 2025

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

FDA Activity Recap: July 2025 Features Multiple CRLs, RMAT Designation, and More

ByNoah Stansfield

August 1st 2025

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

CGTLive®’s Weekly Rewind – August 1, 2025

ByNoah Stansfield

August 1st 2025

Review top news and interview highlights from the week ending August 1, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – July 30, 2025

ByNoah Stansfield

July 30th 2025

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

ImmunoLogic, Episode 5: "Tumor Infiltrators and Triumphs: The Evolution of TIL Therapy" With Michael T. Lotze, MD

ByJoseph A. Fraietta, PhD,Michael T. Lotze, MD,Noah Stansfield,Matt Hoffman

July 30th 2025

In episode 5 of ImmunoLogic, Michael T. Lotze, MD, discusses the evolution and future of tumor-infiltrating lymphocyte therapies.

CGTLive®’s Weekly Rewind – July 25, 2025

ByNoah Stansfield

July 25th 2025

Review top news and interview highlights from the week ending July 25, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – July 23, 2025

ByNoah Stansfield

July 23rd 2025

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

CGTLive®’s Weekly Rewind – July 18, 2025

ByNoah Stansfield

July 18th 2025

Review top news and interview highlights from the week ending July 18, 2025.

Five Advanced Therapeutics to Watch in 2H 2025

ByNoah Stansfield

July 17th 2025

The CGTLive® team highlights 5 therapeutics that are nearing major decisions by the FDA.

Around the Helix: Cell and Gene Therapy Company Updates – July 16, 2025

ByNoah Stansfield

July 16th 2025

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

Ultragenyx’s MPS IIIA Gene Therapy UX111 Met With CRL

ByNoah Stansfield

July 14th 2025

According to Ultragenyx, the CRL relates to a need for additional CMC information and improvements and observations from inspections of manufacturing facilities.