Movement Disorders

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According to the company, VQ-101 is the first small molecule to show over 75% activation of the lysosomal enzyme GCase in humans, with phase 1 results in healthy volunteers indicating tolerability and exposure levels that support once-daily dosing.

After just 6 weeks of treatment, patients showed antibody levels 16 times higher than those in the placebo group, highlighting the vaccine’s potential to slow disease progression.

Initial findings showed significant improvements from baseline in the disease-specific, patient-reported quality of life scores in both the high and low dose AAV-GAD groups, with no significant change in the sham group at 26 weeks.

A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.

The phase 1 study, which is currently recruiting, includes a 4-week screening period, a 52-week treatment period, and a 4-year long-term extension to continue to test the effects of HG202.

Here's some of what is coming soon to NeurologyLive® this week.

Catch up on any of the neurology news headlines you may have missed over the course of October 2024, compiled all into one place by the NeurologyLive® team.

Diego Torres-Russotto, MD, chair of neurology at Baptist Health Miami Neuroscience Institute, discussed the Miami Neuroscience Symposium’s focus on addressing the community’s neurological needs, balancing content delivery and fostering multidisciplinary collaboration.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

Michael McDermott, MD, chief medical executive at Baptist Health Miami Neuroscience Institute, gave greater insights on the institute’s annual symposium, which offers attendees updates in cutting-edge imaging, surgical techniques, and neurocognitive research.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on GBA1-associated Parkinson disease.

As part of our monthly clinician spotlight, NeurologyLive® highlighted movement disorder expert Sana Aslam, DO, an assistant professor of neurology at University of Colorado School of Medicine.

Here's some of what is coming soon to NeurologyLive® this week.

CAP-003, a non-invasive gene therapy, exceeded the 30% efficacy threshold for normalizing GCase activity in patients across all doses observed.

David Devos, MD, PhD, a neuropharmacologist at the University of Lille, in France, discussed positive data from an early-stage study assessing InBrain Pharma’s device-assisted therapy in Parkinson disease.

Based on the positive data, the company plans to begin a phase 3 trial assessing FLT203 in patients with Gaucher disease type 1 in the second half of 2025.

Here's some of what is coming soon to NeurologyLive® this week.

In clinical trials, patients with Parkinson disease who were treated with foscarbidopa/foslevodopa experienced superior improvement in ON time without dyskinesia compared with oral immediate-release carbidopa/levodopa.

Jonas Hannestad, MD, PhD, chief medical officer at Gain Therapeutics, provided clinical perspective on promising data from a phase 1 first-in-human study of GT-02287, an investigational therapy for Parkinson disease with or without a GBA1 mutation.

Here's some of what is coming soon to NeurologyLive® this week.

Catch up on any of the neurology news headlines you may have missed over the course of September 2024, compiled all into one place by the NeurologyLive® team.

The orphan medicinal product designation given by the European Commission was based on findings produced in research funded by the United States Department of Defense.

Over a 12-month period, patients showed sustained improvements in disease progression through valbenazine, regardless of concomitant antipsychotic treatment.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending October 4, 2024.

The KINETIC 2 study did not show statistically significant improvement in essential tremor symptoms compared to placebo, leading to the termination of its development for this condition.

This trial aims to explore the role of the noradrenergic system in behavioral and cognitive outcomes and could lead to new therapeutic strategies for managing PSP.

ION464 is designed to inhibit the production of the alpha-synuclein protein, aiming to reduce the accumulation of this protein in the brain, which is believed to be a key factor driving neurodegeneration in MSA and Parkinson disease.

The consultant neurologist and senior lecturer at St. Georges Hospital in London, provided commentary on the diagnosis, management, and future research around neuropsychiatric symptoms of Parkinson disease.