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Lysosomal Disorders

Latest News

August 24th 2026
PTC Wins Sangamo's Fabry Gene Therapy in Bankruptcy Auction

PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.

August 20th 2026

Pariglasgene Brecaparvovec-opnr Approved for Glycogen Storage Disease 1a

May 27th 2026

Around the Helix: Cell and Gene Therapy Company Updates – May 27, 2026

May 20th 2026

Around the Helix: Cell and Gene Therapy Company Updates – May 20, 2026

May 13th 2026

Around the Helix: Cell and Gene Therapy Company Updates – May 13, 2026

Latest Videos

Podcasts

ImmunoLogic Episode 9
ImmunoLogic, Episode 9: "Rewiring Immunotherapy for High-Risk Neuroblastoma" With Kristopher R. Bosse, MD
ImmunoLogic Episode 8
ImmunoLogic, Episode 8: "Surgery Meets Immunotherapy: A One-Two Punch for Breast Cancer" With Julia C. Tchou, MD, PhD, FACS
ImmunoLogic Bonus Episode
ImmunoLogic, Bonus Episode: "Chemo Optional, Data Mandatory - A Year-End Recap"
ImmunoLogic Bonus Episode
ImmunoLogic, Bonus Episode: "ASH 2025: Practice-Changers on the Ground and on the Wi‑Fi"
ImmunoLogic Episode 7
ImmunoLogic, Episode 7: "Turbocharging CAR T-Cells: The IL-9 Orthogonal Signal" With Anusha Kalbasi, MD
Special Report Episode 10
Quarter Century Update: The Impact of Artificial Intelligence
Special Report Episode 9
Quarter Century Update: Thoughts on Reducing Costs of Goods
Special Report Episode 7
Quarter Century Update: Future Predictions for the Field
Special Report Episode 7
Quarter Century Update: What Does the Field Need Most Right Now?
Special Report Episode 6
Quarter Century Update: What Lessons Should Guide the Next Generation of Developers and Regulators?

CME Content

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Cases & Conversations: Real-World Insights on the Diagnosis and Management of Cold Agglutinin Disease
Virtual Event

Cases & Conversations: Real-World Insights on the Diagnosis and Management of Cold Agglutinin Disease

September 2, 2026

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(CME Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Kentucky
Video

(CME Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Kentucky

Lori A. Leslie, MD; Sushil Bhardwaj, MD, FACP

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Houston Hematology Satellite Symposia
In-Person + Virtual Event

Houston Hematology Satellite Symposia

September 10, 2026

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Arkansas
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Arkansas

Lori A. Leslie, MD; Alex Mejia, MD

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – New York
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – New York

Lori A. Leslie, MD; Bachar Samra, MD

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Tennessee
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Tennessee

Krish Patel, MD; Premila Malhotra, MD

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – North Carolina
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – North Carolina

Krish Patel, MD; Tara Graff, DO, MS

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Montana/Wyoming
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Montana/Wyoming

Lori A. Leslie, MD; Jack W. Erter III, MD, MMHC

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Washington
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Washington

Krish Patel, MD; Girish Kunapareddy, MD

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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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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Nevada
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Nevada

Krish Patel, MD; Matthew Ulrickson, MD

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(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Kentucky
Video

(CPE Track) Community Oncology Connections™: Leveraging Recent Advances in the Treatment of Relapsed/Refractory Follicular Lymphoma – Kentucky

Lori A. Leslie, MD; Sushil Bhardwaj, MD, FACP

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

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

Claire Roddie, MD, PhD; Elias Jabbour, MD; Hagop Kantarjian, MD

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Community Practice Connections™ Next Generation Therapies in AML: Menin Inhibitors, IDH1/2, Combinations, and Beyond!
Multimedia

Community Practice Connections™ Next Generation Therapies in AML: Menin Inhibitors, IDH1/2, Combinations, and Beyond!

Naval Daver, MD

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(CME Track) Tackling Oncologic Emergencies in Patients Treated With High-Dose Methotrexate
Video

(CME Track) Tackling Oncologic Emergencies in Patients Treated With High-Dose Methotrexate

Shazia Nakhoda, MD; Shruti Gupta, MD, MPH; Anitha Varghese, MSN, APRN, AGPCNP-C

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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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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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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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Real-World Insights on Advances in the Management of Myeloproliferative Neoplasms: How the Experts Apply the Latest Developments to Clinical Practice
Video

Real-World Insights on Advances in the Management of Myeloproliferative Neoplasms: How the Experts Apply the Latest Developments to Clinical Practice

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

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