Lysosomal Disorders

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Latest Videos

Joseph Rossano, MD, on the Potential of Gene Therapy in Danon Disease

Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare Disorders

Improving Access to Rare Disease Care

Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health Care

Devon J. Shedlock, PhD, on Shifting from Autologous to Allogeneic Therapies

Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2

Treating Autoimmune Diseases With Smarter Cell Therapies

Working With the Community to Improve Patient Access to Trials, Therapies

Roberto Giugliani, MD, PhD, on Reducing GAGs in MPS Type 2 With Gene Therapy

Paul Wuh-Liang Hwu, MD, PhD, on Questions Remaining With AADC Deficiency

CME Content

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

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

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

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

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

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

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

Video

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

Krish Patel, MD; Girish Kunapareddy, MD

Case-based Simulation

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

Jae Park, MD

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

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

Video

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

Krish Patel, MD; Matthew Ulrickson, MD

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

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

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

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

Multimedia

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

Naval Daver, MD

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

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

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

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

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

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

Adaptive Learning

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

Video

(CME Track) Medical Crossfire®: Menin Inhibitors in AML—Dissecting the Data to Define the Role

Naval Daver, MD; Courtney DiNardo, MD; Eunice Wang, MD

Adaptive Learning

Show Me the Data®: How Today’s Evidence Is Shaping Tomorrow’s Management and Prophylaxis of Chronic GVHD

Carrie L. Kitko, MD; Miguel-Angel Perales, MD; Amandeep Salhotra, MD

Video

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

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

Video

Oncology Town Hall: Translating the Latest Updates Into the Care of Patients With AML in Community Practice

Eunice Wang, MD; Hetty Carraway, MD, MBA; Naval G. Daver, MD

More News

Around the Helix: Cell and Gene Therapy Company Updates – March 19, 2025

ByNoah Stansfield

March 19th 2025

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

CGTLive®’s Weekly Rewind – March 14, 2025

ByNoah Stansfield

March 14th 2025

Review top news and interview highlights from the week ending March 14, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – March 12, 2025

ByNoah Stansfield

March 12th 2025

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

David-Alexandre C. Gros, MD, Eledon’s chief executive officer

Addressing Unmet Needs in End Stage Kidney Disease With Genetically Modified Porcine Kidney Transplant

ByDavid-Alexandre C. Gros, MD,Noah Stansfield

March 12th 2025

David-Alexandre Gros, MD, the chief executive officer of Eledon Pharmaceuticals, discussed the company’s collaboration with EGenesis.

ImmunoLogic, Episode 2: "Charting New Frontiers in CAR T-Cell Safety" With Bruce Levine, PhD

ByJoseph A. Fraietta, PhD,Janna Minehart, MD,Noah Stansfield,Matt Hoffman

March 11th 2025

In Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.

CGTLive®’s Weekly Rewind – March 7, 2025

ByNoah Stansfield

March 7th 2025

Review top news and interview highlights from the week ending March 7, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – March 5, 2025

ByNoah Stansfield

March 5th 2025

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

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital

Future Plans for Evaluating Spur Therapeutics’ FLT201

ByReena Sharma, MD,Noah Stansfield

March 5th 2025

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.

CGTLive®’s Weekly Rewind – February 28, 2025

ByNoah Stansfield

February 28th 2025

Review top news and interview highlights from the week ending February 28, 2025.

Around the Helix: Cell and Gene Therapy Company Updates – February 26, 2025

ByNoah Stansfield

February 26th 2025

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

Reena Sharma, MD, on Future Plans for Evaluating FLT201

ByReena Sharma, MD,Noah Stansfield

February 23rd 2025

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

CGTLive®’s Weekly Rewind – February 21, 2025

ByNoah Stansfield

February 21st 2025

Review top news and interview highlights from the week ending February 21, 2025.

FDA Accepts BLA for Ultragenyx’s MPSIIIA Gene Therapy UX111 With Priority Review

ByNoah Stansfield

February 19th 2025

The PDUFA action date for the BLA has been set at August 18, 2025, and no advisory committee meeting is currently planned.

Around the Helix: Cell and Gene Therapy Company Updates – February 19, 2025

ByNoah Stansfield

February 19th 2025

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

Assessing Spur Therapeutics’ Gene Therapy FLT201 in Gaucher Disease

ByReena Sharma, MD,Noah Stansfield

February 17th 2025

Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed results she presented at the 21st Annual WORLDSymposium.

CGTLive®’s Weekly Rewind – February 14, 2025

ByNoah Stansfield

February 14th 2025

Review top news and interview highlights from the week ending February 14, 2025.

Reena Sharma, MD, on Assessing Spur Therapeutics’ Gene Therapy FLT201 in Gaucher Disease

ByReena Sharma, MD,Noah Stansfield

February 13th 2025

The adult metabolic consultant at Salford Royal Hospital discussed results she presented at the 21st Annual WORLDSymposium.

Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025

ByNoah Stansfield

February 12th 2025

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

CGTLive®’s Weekly Rewind – February 7, 2025

ByNoah Stansfield

February 7th 2025

Review top news and interview highlights from the week ending February 7, 2025.

Updated Data Suggests Sangamo’s Gene Therapy ST-920 Improves Renal Function in Fabry Disease

ByNoah Stansfield

February 6th 2025

A positive mean eGFR slope was seen in 23 patients who reached 1 year of follow-up in the phase 1/2 STAAR trial.

Eric Crombez, MD, the chief medical officer at Ultragenyx

Ultragenyx’s Gene Therapy UX111 Improves Clinical Function in MPSIII

ByNoah Stansfield

February 6th 2025

With regard to safety, Ultragenyx characterized the gene therapy as “generally well-tolerated."

Around the Helix: Cell and Gene Therapy Company Updates – February 5, 2025

ByNoah Stansfield

February 5th 2025

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

Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Continues to Show Safety and Efficacy in Phase 1/2 Trial

ByNoah Stansfield

February 4th 2025

Among the 5 patients in the efficacy set, 4 patients ceased receiving their previous standard of care therapy.

CGTLive®’s Weekly Rewind – January 31, 2025

ByNoah Stansfield

January 31st 2025

Review top news and interview highlights from the week ending January 31, 2025.

ImmunoLogic, Episode 1: "Chemo-Free Lymphoma? Seriously?" With Stephen J. Schuster, MD

ByJoseph A. Fraietta, PhD,Janna Minehart, MD,Noah Stansfield,Matt Hoffman

January 31st 2025

In Episode 1 of ImmunoLogic, Stephen J. Schuster, MD, discussed the potential of treating lymphoma without chemotherapy.