Neurology

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

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

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

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

Review some of our most-viewed coverage of advancements in cell therapies, including the latest study results and insights from experts in the field.

Review top news and interview highlights from the week ending December 24, 2021.

The program is still on clinical hold following SUSARs of myelodysplastic syndrome in treated participants.

The FDA has placed the investigational new drug application for the treatment on clinical hold.

Review top news and interview highlights from the week ending December 17, 2021.

Review top news and interview highlights from the week ending December 9, 2021.

Autologous adipose stem cell data in a small cohort provides a feasibility for future validation studies.

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, trial data, and further research.

The professor from National Taiwan University Hospital discussed gene therapy studies in AADC deficiency, Parkinson disease, SMA, and Pompe disease.

Review top news and interview highlights from the week ending December 3, 2021.

The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.

ALSFRS-R and FVC values were stable 3 months after treatment but significantly decreased 6 months after treatment, warranting further investigation.

Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the advantages of developing gene therapies for both dogs and humans.

Review top news and interview highlights from the week ending November 26, 2021.

The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.

From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.

Review top news and interview highlights from the week ending November 19, 2021.

The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.