Neurology

Review top news and interview highlights from the week ending October 4, 2024.

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

Daniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.

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

CSF analyses from 7 patients treated in the study showed reductions of more than 80% in NAA levels from baseline.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The company pointed out that today, September 30, is LGMD Awareness Day.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.

Review top news and interview highlights from the week ending September 27, 2024.

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

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.

In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.

Review top news and interview highlights from the week ending September 20, 2024.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, shared his outlook on the trajectory of research in the field.

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

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, highlighted studies presented at MDA's 2024 conference.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed his research career with muscular dystrophies.

Review top news and interview highlights from the week ending September 13, 2024.

Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

The gene therapy is currently being evaluated in the phase 1/2 CANaspire clinical trial.

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

Biogen plans to submit for regulatory approval for a higher dose based on the positive topline data from the phase 2/3 DEVOTE study (NCT04089566).

In honor of World Duchenne Awareness Day, held September 7, several experts have provided their insights on the real-world experience of gene therapy’s use in Duchenne muscular dystrophy.

Review top news and interview highlights from the week ending September 6, 2024.

The data come from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial.

Notably, the therapy previously received rare pediatric disease designation from the FDA in July 2024.

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 FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The therapy is being developed for the treatment of patients with progressive MS who have imaging evidence of ongoing inflammatory tissue injury and who are HLA-DRB1*15:01 positive.