Neurology

The CGTLive^™ Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. It also contains up-to-date clinical news coverage in the field of gene and cell therapy for central nervous system disorders.

Latest News

FDA Activity Recap: March 2025 Features FDA Approvals in Hemophilia, ATTR Amyloidosis, and More

By Noah Stansfield

April 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.

Robert Califf, MD, MACC, a cardiologist and former FDA commissioner

Urgency of Funding and Equity in Neuromuscular Research and Care

By Robert M Califf, MD, MACC
Noah Stansfield

March 31st 2025

Natalie Goedeker, CPNP, the codirector of pediatric neuromuscular clinical research at Washington University in St. Louis

Handling Neuromuscular Gene Therapy at Real-World Sites

By Natalie Goedeker, CPNP
Noah Stansfield

March 30th 2025

CGTLive®’s Weekly Rewind – March 28, 2025

By Noah Stansfield

March 28th 2025

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

By Noah Stansfield

March 26th 2025

Latest CME Events & Activities

Fighting Disparities and Saving Lives: An Exploration of Challenges and Solutions in Cancer Care

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

More News

CGTLive’s Weekly Rewind – February 18, 2022

GeneTherapyLive Staff

February 18th 2022

Review top news and interview highlights from the week ending February 18, 2022.

Gene Therapy Shows Potential in Canavan Disease

Victoria Johnson

February 17th 2022

Myrtelle’s therapy targets oligodendrocytes to deliver a functional copy of the ASPA gene.

Elucidating Associations Between EBV and MS: Jakob Dupont, MD

Jakob Dupont, MD

February 17th 2022

The global head of research and development at Atara Biotherapeutics discussed the link between EBV and MS.

Around the Helix: Cell and Gene Therapy Company Updates - February 16, 2022

Victoria Johnson

February 16th 2022

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

Gene Therapy for Friedreich Ataxia Cardiomyopathy Set to Move Forward

Alicia Bigica

February 16th 2022

The FDA has approved LEXEO Therapeutics’ investigational new drug application.

Gene Therapy Well-Tolerated in Late-Onset Pompe Disease

Abby Reinhard

February 13th 2022

Interim safety data from the FORTIS study were presented at WORLDSympsoium.

Fibroblast Cell Therapy or Stem Cell Therapy for MS: Hamid Khoja, PhD

Hamid Khoja, PhD

February 11th 2022

The chief scientific officer of FibroBiologics discussed the advantages of fibroblast cell therapy over stem cell therapy.

Gene Therapy Stabilizes Disease Progression in GM1 Gangliosidosis

Victoria Johnson

February 10th 2022

Positive results from a phase 1/2 study of AXO-AAV-GM1 were presented at WORLDSymposium.

Around the Helix: Cell and Gene Therapy Company Updates - February 9, 2022

Victoria Johnson

February 9th 2022

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

Around the Helix: Gene and Cell Therapy Company Updates - February 2, 2022

Victoria Johnson

February 2nd 2022

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

IND Cleared for Adrenomyeloneuropathy Gene Therapy

Victoria Johnson

February 2nd 2022

SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.

Giant Axonal Neuropathy Gene Therapy Well-Tolerated in High-Dose Cohort

Victoria Johnson

January 31st 2022

The positive news is countered by a report of a patient death in Taysha’s gene therapy program for GM2 gangliosidosis.

GeneTherapyLive’s Weekly Rewind – January 28, 2022

GeneTherapyLive Staff

January 28th 2022

Review top news and interview highlights from the week ending January 28, 2022.

Around the Helix: Gene and Cell Therapy Company Updates - January 26, 2022

Victoria Johnson

January 26th 2022

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

Stem Cell Gene Therapy Efficacious in Metachromatic Leukodystrophy

Giuliana Grossi

January 25th 2022

Investigators from IRCCS San Raffaele analyzed results from a phase 1/2 study on atidarsagene autotemcel.

Around the Helix: Gene and Cell Therapy Company Updates - January 19, 2022

Victoria Johnson

January 19th 2022

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

FDA Extends BLA Review for bluebird bio’s β-Thalassemia and CALD Gene Therapies

Victoria Johnson

January 18th 2022

Eli-cel remains on clinical hold in cerebral adrenoleukodystrophy after a report of persistent anemia.

GeneTherapyLive’s Weekly Rewind – January 14, 2022

GeneTherapyLive Staff

January 14th 2022

Review top news and interview highlights from the week ending January 14, 2022.

Around the Helix: Gene and Cell Therapy Company Updates

Victoria Johnson

January 11th 2022

Many cell and gene therapy companies are initiating new programs and collaborations with the start of the new year.

Gene Therapy Continues to Show Efficacy in Duchenne Muscular Dystrophy

Victoria Johnson

January 11th 2022

The placebo crossover cohort experienced statistically significant improvements in NSAA score over external control.

GeneTherapyLive’s Weekly Rewind – January 7, 2022

GeneTherapyLive Staff

January 7th 2022

Review top news and interview highlights from the week ending January 7, 2022.

Angelman Syndrome Gene Therapy Well Tolerated

Victoria Johnson

January 6th 2022

The DSMB has recommended that enrollment expand and doses be escalated from 3.3 to 5 mg in 1 cohort of the KIK-AS study.

AMT-130 for Huntington Disease Well-Tolerated

Matt Hoffman

January 4th 2022

Data from a phase 1/2 trial of AMT-130 supports continued enrollment for a higher-dose cohort of 6 x 1013 vg.

Strategies to Reverse Multiple Sclerosis Neurodegeneration: Robert Zivadinov, MD, PhD

Robert Zivadinov, MD, PhD

December 30th 2021

The director of the Buffalo Neuroimaging Analysis Center and professor of Universtiy of Buffalo discussed avenues of research into reversing neurodegeneration in MS.