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Parkinson Agent HL192 Successfully Completes Phase 1 First-In-Human Study

Hearing Loss Associated With Parkinson Disease Diagnosis, Large-Scale Study Shows
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Findings from a recently published study suggest patients with Parkinson disease can reduce postural sway as efficiently as neurologically healthy patients when performing horizontal saccades.

At week 10, investigators observed no differences between triheptanoin and placebo on the frequency of movement disorder events in patients with Glut1 deficiency disorder.

The global, placebo-controlled, 12-week study aims to enroll 330 participants and expects topline results in the first half of 2026.

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.

The doctor of nursing practice specialized in movement disorders discussed specific data the StrivePD app captures and how it can enhance quality of life for patients with Parkinson disease. [WATCH TIME: 3 minutes]

The doctor of nursing practice specialized in movement disorders provided clinical insight on how the StrivePD application empowers patients with Parkinson disease to track symptoms, medication, and daily activities using an iPhone and Apple Watch. [WATCH TIME: 3 minutes]

The consultant neurologist and senior lecturer at St. Georges Hospital in London, explores the origins of common neuropsychiatric symptoms experienced by patients with Parkinson disease and how they may be exasperated. [WATCH TIME: 4 minutes]

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.

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.

FDA Approves AbbVie’s 24-Hour Foscarbidopa/Foslevodopa Pump for Advanced Parkinson Disease Treatment
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.

The chief medical officer and chief development officer at BlueRock Therapeutics discussed the promise behind bemdaneprocel and its unique mechanism of action relative to other Parkinson disease treatments. [WATCH TIME: 3 minutes]

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.

A recent survey showed that patients with Parkinson disease quickly adapted to using continuous subcutaneous apomorphine infusion therapy, with high amount of recommending the device to others.

Amantadine extended-release capsules, approved for levodopa-related dyskinesia and OFF episodes in Parkinson disease, offer high amantadine concentrations that peak in the time of first morning levodopa dose.

The chief medical officer and chief development officer at BlueRock Therapeutics provided perspective on newly presented data investigating a cell therapy for patients with Parkinson disease. [WATCH TIME: 4 minutes]

The president and chief executive officer at Praxis Precision Medicine provided clinical insight on the promising build of ulixacaltamide, an investigational, selective agent in development for essential tremor. [WATCH TIME: 3 minutes]

ELATE, a multicenter, randomized, double-blind, placebo-controlled trial, aims to address the limitations of current oral therapies by exploring a targeted, localized treatment option.

In a post hoc analysis of the COSMOS study, 24-hour LCIG infusion led to a reduction in nocturnal and morning akinesia, though many patients continued to rely on nighttime add-on medications.

The neuropharmacologist at the University of Lille, in France, provided commentary on promising results from the phase 1/2 DIVE-I trial assessing the safety and efficacy of a device-assisted therapy in Parkinson disease. [WATCH TIME: 6 minutes]

While participants responded positively to nutritional guidance and the Mediterranean diet, they expressed negative views on the ketogenic diet due to its restrictive nature, social impracticality, and gastrointestinal side effects of MCT oil.