Marco Meglio

In preclinical mouse models lacking both dystrophin and utrophin, treatment with an agent using the company's PN chemistry resulted in 100% survival at 40 weeks, setting up the therapy for this phase 1b2/a trial.

After 10-12 months of treatment, 51.2% of patients responded with a clinically meaningful reduction in drop seizures, while 25.3% of patients demonstrated a profound reduction.

Neurology News Network for the week of October 2, 2021. [WATCH TIME: 4 minutes]

David R. Lynch, MD, PhD, professor of neurology, University of Pennsylvania Perelman School of Medicine, discussed why omaveloxolone’s recent success speaks to the progress made within the FA treatment space.

Children and adults with migraine displayed significantly less REM sleep as a percentage of total sleep time than healthy individuals.

Even after excluding those who received ALS diagnosis in the 5 years prior, higher HDL and APOA1 levels were associated with a lower risk of ALS.

The multinational study uses change from baseline to week 12 in total Unified Parkinson’s Disease Rating Scale score, defined as sum of parts II and III scores, as the primary end point.

The agent had previously demonstrated positive results in a phase 2a setting, with favorable trends on modified Unified MSA Rating Scale scores across a 12-week period.

The professor of neurology at the University of Pennsylvania Perelman School of Medicine discussed the state of care for Friedrich ataxia and omaveloxolone’s potential to become its first approved therapy.

The submission includes findings from Study 1601, along with long-term safety and efficacy data from Zogenix’s ongoing open-label extension trials.

Despite not achieving statistical significance, there was greater relative reduction in seizure frequency over placebo in those with a structural etiology for focal epilepsy than those without, laying the foundation for future research.

Jessica Caldwell, PhD, director, Women’s Movement Prevention Center, Cleveland Clinic, sat down shed light on the understanding of Alzheimer disease in women and their inclusion in clinical trials.

Between PREVENT baseline and end, mean EDSS and Hauser Ambulation Index scores improved with eculizumab (Soliris; Alexion) monotherapy and deteriorated with placebo alone.

The findings identified Th2-related cytokines as characterizing for the prognosis of acute episodes of NMOSD at 1 month and found serum NfL to be a likely biomarker of disease severity at attack.

The expanded access program is designed to provide individuals with ALS who are not eligible to enroll in the Healey ALS Platform Trial access to CNM-Au8.

More than 80% of those with NMOSD experienced disease relapse 6 months after discontinuation of immunosuppressive therapy.

On World Narcolepsy Day 2021, get a recap of the latest advances in narcolepsy that you might have missed over the last few months, compiled all into one place by the NeurologyLive team.

The director of the Women’s Movement Prevention Center at Cleveland Clinic discussed her new study, and the current knowledge of the role genetics play in Alzheimer disease risk.

Over a 12-month stretch, patients with spinocerebellar ataxia type 2 treated with riluzole showed significant worsening in Composite Cerebellar Functional Severity Score.

Both the 200-mg and 350-mg dose groups showed numerically advantageous differences over placebo in secondary end points such as average daily pain score and 30% reduction in ADP.

Patients previously randomized to omaveloxolone in the core study period continued to show no worsening on modified Friedrich Ataxia Rating Scale relative to their original baseline after nearly 2.5 years of treatment.

Despite not meeting the primary end point, those in the early-start group showed reduced change from baseline in MDS-UPDRS-III scores compared with the delayed-start group at both weeks 52 and 104.

Neurology News Network for the week ending September 18, 2021.

Improvements in mean total amount of time and percentage of participants that were awake suggested limited effect with opicapone (Ogentys; Neurocrine Bio).

Patients with Parkinson disease in both the 24-hour and 16-hour ND0612 dosing regimen groups had ON-state UPDRS-motor scores and UPDRS-ADL scores improve over the 12-month period.

Torticollis was the most common predominant cervical dystonia presentation subtype at injection 1, followed by laterocollis.

When comparing only the lowest and highest HbA1c groups with each other, unadjusted and adjusted regression analysis indicated faster motor progression in the highest group.

The director of IT and Neuroinformatics Development at the Buffalo Neuroimaging provided thoughts on artificial intelligence’s role in neuroimaging and treating multiple sclerosis.

After receiving expanded indication from the FDA as both an acute and preventive treatment of cluster headache, gammaCore becomes the first treatment for paroxysmal hemicrania and hemicrania continua.

The SELECT-HD trial (NCT05032196) is expected to enroll approximately 36 patients with a confirmed diagnosis of early-stage Huntington disease and carry the SNP3 allele in association with their CAG expression.