Neurology

The data come from patients treated across 2 studies.

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

A recent patient death following treatment with the gene therapy raised concerns for the European Medicines Agency.

Review top news and interview highlights from the week ending April 4, 2025.

The move was made in relation to the previously-announced death of a US teenager who received the therapy.

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

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

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, discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.

Natalie Goedeker, CPNP, the codirector of pediatric neuromuscular clinical research at Washington University in St. Louis, discussed the session she chaired at MDA’s 2025 conference.

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

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

In pooled data from 156 patients, there were no deaths or study discontinuations.

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, discussed his powerful journey as a patient advocate and gene therapy pioneer, shedding light on challenges and progress in LGMD.

Michael Flanagan, PhD, the chief scientific officer at Avidity, discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

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

In observance of Multiple Sclerosis Awareness Month, held annually in March, we took a look back at the past year's news in cell therapy for MS.

One of the patients, who was aged 3 years at the time of treatment, achieved 122.3% microdystrophin expression compared to control.

Patients treated at Dose 2 in the dose-escalation phase of Part 1 of the study showed a persistent 68% reduction in CK levels over 2 years.

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

OAV101 IT is a version of Zolgensma that is delivered directly to the spine.

Acute liver injury is known to be a possible adverse event associated with AAV vector-based gene therapies such as Elevidys.

The findings were presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

Adverse events related to the gene therapy itself were mild or moderate, with most occurring in the first 90 days posttreatment.

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

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.

The study included 48 patients, 36 of whom received laromestrocel and 12 of whom received a placebo.

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

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

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.