Marco Meglio

In preclinical models, treatment with GT-02287 restored GCase enzymatic function, reduced aggregated a-synuclein, neuroinflammation and neuronal death, increased dopamine levels, and improved motor function.

In a phase 3 study, patisiran met its primary and first secondary end point, demonstrating significant benefit in functional capacity, quality of life, and health status, in comparison with placebo.

After 1 year of treatment with risdiplam, 81% of the 26-infant cohort were able to sit independently for 30 seconds, a milestone not typically reached in SMA.

Michael Grandner, PhD, associate professor of psychiatry at the University of Arizona College of Medicine, discussed Sleep Reset, an app-based, personalized sleep program that uses sleep coaches to help guide patients in the care of their sleep disorders.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Gunter Hoglinger, MD. [LISTEN TIME: 14 minutes]

The phase 3 study is expected to include 86 children with ataxia-telangiectasia who will be assessed on changes in a rescored modified International Cooperative Ataxia Rating Scale.

During the week after emergency department discharge, both low- and high-dose participants reported a median of 2 headache days, signifying little difference.

In a cohort of moderately severe Alzheimer disease, patients on placebo demonstrated cognitive decline while those on active treatment showed no significant decline.

Using treatment failures and antiseizure medication retention, findings pointed to levetiracetam as a more effective initial monotherapy in females with juvenile myoclonic epilepsy.

After being diagnosed with acute flaccid myelitis more than 10 years ago, patient advocate Sarah Todd Hammer spoke on the ways in which disability should be viewed and discussed.

At the conclusion of a 12-weel treatment period, biomarker data provided mechanistic insights of senolytic effects using dasatinib and quercetin that need to be confirmed in fully powered, placebo-controlled studies.

The adjusted odds ratios observed for any vaccine and MS flare-ups requiring hospitalization were similar regardless of sex, with the highest odds observed for the pneumococcal vaccine.

Although the mortality among monitored and nonmonitored patients was similar, the monitored group had significantly more patients with severe disability and fewer with good recovery at 6 months.

Previous data on the 25 mg cohort of CTI-1601 showed significant reductions in frataxin levels among treated individuals with Friedreich ataxia.

Years of genetic research has uncovered more about the underpinnings of limb-girdle muscular dystrophy, leading to potential disease-modifying therapies in the near future.

In a phase 3 setting, the 2-component therapy showed positive trends or clinically meaningful improvements on motor and respiratory functions compared with previously approved enzyme replacement therapy.

Over a 4-week treatment period, patients in both Lu AG09222 groups experienced about 2 fewer migraine days per month compared with placebo.

After deliberating, the FDA committee expressed concerns about the mechanism of action of NurOwn, manufacturing details, and consistency of data.

By targeting tau-ack280, ADEL-Y01 inhibits tau aggregation and propagation, and promotes microglial tau clearance.

The chief of the Movement Disorders Division at Mass General Hospital provided context on the goals behind the research careers reimagined course at the recently concluded ANA Annual Meeting.

Virginia Gao, MD, PhD, a movement disorders fellow at Weill Cornell Medicine, discussed several topics related to the use of biomarkers in the prodromal stages of neurodegenerative disorders like Parkinson disease.

In the largest meta-analysis of patients with poststroke seizures, findings showed that those with early seizures had a greater mortality risk than those with late.

In the study, patients with ALS will be asked to complete questionnaires, a physical examination, and undergo neurostimulation site visits 3 times per week for 4 weeks.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Birgitte Kornum, MD. [LISTEN TIME: 18 minutes]

The double-blind trial will feature 180 patients of various spinal muscular atrophy types who will be followed over a 48-week treatment period.

The associate professor in the department of neurology at the University of Rochester provided insight on her presentation on psychosis in Parkinson disease and related disorders given at the 2023 ANA Annual Meeting.

The AIRAscore may help optimize the diagnostic process of neurodegenerative disorders by comparing brain structures with collected age- and gender-specific qualitative data samples.

Approved in 2016, the newly updated label changes add more clarity to the use of pimavanserin, noting that patients with Parkinson disease with dementia may be eligible for treatment.

Visual hypersensitivity decreased after 3 months of treatment, with a clear association with clinical response to treatment regarding migraine days.

Marcio Souza, president and chief executive officer at Praxis Precision Medicines provided follow-up thoughts on recently announced positive findings assessing ulixacaltamide in patients with essential tremor.