Neurology

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

The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.

Wave Life Sciences is discontinuing WVE-004.

Reductions in NfL were also associated with changes on ALSFRS-R scores.

Review top news and interview highlights from the week ending July 7, 2023.

Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.

In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.

The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.

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 June 30, 2023.

Taysha also provided updates on its gene therapy program for Rett syndrome.

Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.

Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

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 June 23, 2023.

Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.

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

uniQure deemed the safety profile for AMT-130 to be manageable.

Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting.

The advocacy groups are funding a 1-year study to be led by Barry Byrne, MD, PhD, at University of Florida.

Review top news and interview highlights from the week ending June 16, 2023.

CGTLive takes a look at challenges that have beset the first gene therapy coming close to market for treating Duchenne muscular dystrophy in 2023.

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 June 9, 2023.

For International Batten Awareness Day, held annually on June 9, CGTLive reached out to several experts to inquire about the potential impact of gene therapy on the Batten disease treatment landscape.