Movement Disorders

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 essential tremor.

Tanya, a patient who lost her mobility due to undiagnosed spinal issues, discussed her life-saving surgery for spinal meningioma, and the complex surgical process she underwent to regain her strength.

Denali Therapeutics plans to seek accelerated FDA approval for DNL310 in Hunter syndrome, showing significant biomarker improvements and clinical benefits in ongoing trials.

Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, talked about how the prioritization of mirdametinib, a promising treatment for both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.

The application is based on results of Prilenia Therapeutics’ phase 3 study that assessed the safety and efficacy of pridopidine in patients with Huntington disease.

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

Mirdametinib, a treatment in development for adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas, was granted a PDUFA target action date of February 28, 2025.

Neurology News Network. for the week ending August 31, 2024. [WATCH TIME: 4 minutes]

The FDA has granted fast track designation to [18F]PI-2620 for 3 neurodegenerative conditions, highlighting its potential to improve early diagnosis and treatment.

Phase 1b trials involving patients with Parkinson are planned for late 2024, with data expected by mid-2025.

Findings from a recent study analyzing 3 cohorts suggested that certain proteins in the cerebrospinal fluid may have potential as biomarkers or therapeutic targets in progressive supranuclear palsy.

EryDex did not meet its primary end point possibly because of delays in treatment reducing the number of patients who received the agent, and different treatment effects based on age.

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

DT-216P2 shows promise in treating patients with Friedreich ataxia based on positive phase 1 trial results that indicated a significant increase in frataxin mRNA levels.

A recent review showed different fluid biomarkers were involved in neurodegeneration, oxidative stress, metabolism, microRNA and novel genes in spinocerebellar ataxia type 3.

Researchers detected annual changes in patients with preataxic and early ataxic spinocerebellar ataxia in brain MRI imaging, clinical scores, gait parameters, and retinal thickness.

According to a recent announcement, Larimar Therapeutics reported that all 7 sites of the open-label extension study were activated for assessing nomlabofusp in Friedreich ataxia.

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

Neurology News Network. for the week ending August 17, 2024. [WATCH TIME: 4 minutes]

The panelists provided clinical insight on the utilization of IPX203, its potential benefits and clinical implications, as well as the practicalities of transitioning patients from other therapies.

The panelists discussed the safety profile of IPX203, considering challenges with transitioning, dosing strategies, and monitoring and adjusting patients’ individual dosage.

Dr. Hauser and Dr. Fernandez provided insight on the body of supportive evidence for IPX203, the notable takeaways from RISE-PD, and the long-term benefits observed from treated patients.

IPX203 combines immediate and extended-release levodopa, utilizing advanced delivery technology to optimize absorption and prolong therapeutic effects in patients with Parkinson disease.

Medtronic's asleep DBS approval could expand treatment options for patients unable to tolerate awake surgery, enhancing access to deep brain stimulation therapy.

In this opening segment, Hauser and Fernandez discussed the FDA approval of IPX203, a new long-acting levodopa formulation for Parkinson disease, highlighting its significance in improving treatment options and patient compliance.