Neurology

No adverse effects on weight, general activity, or survival in knockout mice and wild type littermates were observed throughout the study.

The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.

The partnership will allow for the development of disease-specific AAV vectors that will serve Biogen's current gene therapy pipeline.

The FDA has lifted a clinical hold placed on an IND for VY-HTT01 in Huntington disease, allowing Voyager Therapeutics to initiate a phase 1/2 study for the agent later this year.

In an AAN plenary talk, Mark H. Tuszynski, MD, PhD, detailed the work he and colleagues have done to push stem cell therapy from the lab to the clinic to improve care for spinal cord injury.

The FDA has granted a fast track designation to LX1001 as a potential treatment for adult patients with APOE4-associated Alzheimer disease.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

AAV2 TrkB-2A-mBDNF resulted in early signs of efficacy in preclinical studies of glaucoma and humanized tauopathy, which could be translatable to other neurodegenerative polygenic disorders.

In 2021, Abeona Therapeutics is anticipating to complete enrollment in its phase 3 VIITAL study and additional findings for ABO-102 and ABO-101.

NurOwn was safe and led to improvements in function and cognition at 28 weeks compared with baseline for patients with progressive multiple sclerosis.

SGT-001 elicited promising improvements in functional and biomarker end points for patients with Duchenne muscular dystrophy.

SRP-9003 elicited sustained protein expression in muscle tissue and stabilized North Star Assessment for Dysferlinopathies scores at 2 years in patients with limb-girdle muscular dystrophy Type 2E.

Updated findings from across a number of studies highlighted the significant benefits of onasemnogene abeparvovec for children with spinal muscular atrophy.

Ninety percent of patients with cerebral adrenoleukodystrophy who were treated with elivaldogene autotemcel were alive and free of major functional disabilities at 2-years.

The FDA has granted a fast track designation to PBFT02, PBKR03, and PBGM01, which are intended as treatments for patients with various rare CNS disorders.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Rocket Pharmaceuticals has summarized multiple major milestones for its 5 gene therapies from 2020 and provided a forecast and outlook for what is expected to be a banner year for the organization.

Advancements in gene therapy approaches have opened the door for a glimpse into the future of care for a number of diseases.

FDA advises against submission of NurOwn as a treatment for ALS, based on a lack of statistical significance in phase 3 study.

Masahito Kawabori, MD, PhD, associate professor, Hokkaido University, discussed results of the phase 2 STEMTRA trial.

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses the safety and efficacy of spinal muscular atrophy (SMA) treatments.

Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

Vamshi Rao, MD, attending physician of Neurology at Ann and Robert H. Lurie Children’s Hospital of Chicago, discusses how disease modifying therapies have improved spinal muscular atrophy (SMA) prognosis.

The associate professor at Hokkaido University and investigator of STEMTRA also discussed other efforts in stroke, Alzheimer disease, and Parkinson disease.

The Pfizer candidate PF-06939926 has also received fast track, orphan drug, and rare pediatric disease designations from the FDA.

Minimal fat infiltration was observed on MR images from the SRP-9001 arm compared to participants from the natural history cohort.

Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.