Marco Meglio

The basis for the FDA’s decision came from the phase 3 ADAPT-SC study, in which subcutaneous efgartigimod showed a slightly better ability to reduce immunoglobulin compared with its previously approved intravenous formulation.

In one of the first phase 3 trials of primary mitochondrial myopathy, elamipretide failed to meet its primary end points; however, a subgroup of patients with nuclear DNA defects saw improvement in 6-minute walk test, raising further questions.

Walter Koroshetz, MD, director of the National Institutes of Neurological Disorders and Stroke at the NIH, provided perspective on the advances in migraine treatment, and the next steps in research needed over the coming years.

Data showed similar rates of discontinuation because of adverse events between those with and without CGRP monoclonal antibodies, as well as no serious AEs observed for those on concomitant medication.

Compared with the overall population, outcomes for the coprimary end points of pain freedom and freedom from most bothersome symptom at 2 hours postdose remained superior in rimegepant-treated individuals.

Atogepant 60 mg once daily group had significantly larger reductions in mean monthly migraine days across weeks 1-12 and weeks 9-12, with no statistical difference in treatment-emergent adverse events or all-cause discontinuation.

Demographic factors of male sex, non-White race, and having a primary language other than English were associated with a lower odds of being diagnosed with migraine.

Patients on candesartan saw statistically significant reductions in the mean frequency of monthly headache days, including moderate to severe headache days.

When comparing galcanezumab, fremanezumab, and erenumab, 3 FDA-approved CGRP medications for preventive migraine, time to switch was lowest for erenumab and similar for galcanezumab and fremanezumab.

A reduction in microglial activation was observed on the PET scans of most patients included. A phase 2 trial of the therapy was announced earlier this year and is still on track to begin sometime in the fall of 2023.

Originally approved under the accelerated approval pathway, the promising efficacy observed in the study will prompt a future regulatory submission for traditional.

Over a 39-day period, treatment with SCI-110 resulted an average reduction of 23% in agitation among patients with Alzheimer disease.

The professor of neurology at UMass Chan School of Medicine provided perspective on the benefits of pushing back school start times based on what research has shown.

Robert A. Hauser, MD, MBA, director of the Parkinson’s and Movement Disorders Center at the University of South Florida, provided insight on the therapeutic potential of IPX203, and why it adds flexibility to the treatment of Parkinson disease.

In a secondary analysis of a double-blind trial, mazindol extended-release outperformed placebo on clinician and patient scales of cataplexy severity and excessive daytime sleepiness.

John Harsh, PhD, clinical research director, Colorado Sleep Institute, sat down at SLEEP 2023 to discuss the RESTORE study results, and why patients with narcolepsy appear to prefer once-nightly sodium oxybate.

The hold comes nearly a month after Health Canada sent PepGen a No Objection Letter to start its phase 2 CONNECT1-EDO51 trial in patients with Duchenne muscular dystrophy.

After discussions with the FDA, the study will assess 2 co-primary end points of change in Rett Syndrome Behavior Questionnaire total score and Clinical Global Impression Improvement Scale score.

Patients on twice-nightly oxybate reported more issues with inconvenience, anxiety, and feeling somewhat, quite a bit, or extremely groggy/unsteady the next morning.

At the conclusion of the analysis, lemborexant was shown to be safe, with significant improvements seen in REM latency, total REM sleep, and other measures.

The chief medical officer of Harmony Biosciences provided perspective on a proof-of-concept study assessing pitolisant’s (Wakix) clinical benefit in reducing excessive daytime sleepiness individuals with Prader-Willi syndrome.

At the conclusion of the 6-week treatment period, preliminary analyses suggested a greater trend toward improvement on subjective measures of insomnia compared with objective sleep measures.

More than 25% of patients in the pooled meta-analysis developed OSA after undergoing vagus nerve stimulation treatment, prompting the need for routine screening for the condition.

Nearly all patients showed improvement on the Patient Global Impression of Change scale, regardless of sleep inertia status, assessed through a visual analog scale.

Following 13 weeks of treatment with once-nightly sodium oxybate, some patients in the 7.5 g and 9.0 g groups showed complete resolution of cataplexy attacks.

PUMAS significantly alleviated depression relative to controls whereas cognitive behavioral therapy-insomnia did not differ from either group.

Among patients with post-acute sequelae from COVID-19 infection, self-reported cognitive symptoms were correlated with severity of fatigue, anxiety, and depression, but not subjective sleep disturbance.

In a study of 59 individuals with obstructive sleep apnea, solriamfetol yielded cognitive improvements at post-dose time points throughout the day, along with improvements in Patient Global Impression of Severity.

Neurofilament light, a biomarker elevated in neurodegeneration and dementia, was higher among poor sleepers with Pittsburgh Sleep Quality Index scores of less than 5.

Most patients completed at least 3 years of treatment, with no new safety signals observed and a treatment discontinuation rate of 5.3%.