Marco Meglio

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided perspective on the phase 4 RESPOND study assessing nusinersen in individuals with SMA who already received gene therapy.

At month 2 of a 3-month treatment period, investigators observed an improvement of 2.75 days favoring tricaprilin.

Accounting for baseline disease covariates, NurOwn-treated participants had reduced neurofilament light values from baseline to week 20 compared with placebo (P <.05).

Interrater agreement between SCORE-AI and human experts was notable, with almost perfect agreement for generalized epileptiform abnormalities and substantial agreement for focal epileptiform discharges, among other findings.

Higher levels of the n-3 fatty acid eicosapentaenoic acid and the n-6 fatty acid linoleic acid were associated with a lower risk of death during follow-up.

In a cohort of patients who experienced 4-14 migraine days/month, treatment with atogepant continued to improve patients’ migraine status regardless of prior medication failures.

Building on positive 1-year data, 24-month data showed statistically significant differences in the Performance of the Upper Limb scale in treated patients in the open-label extension relative to those on placebo in the double-blind period.

On average, younger and older participants who were unable to sit without support improved from baseline on CHOP-INTEND scores over a 183-day period.

Over a 96-week period, more older patients with relapsing multiple sclerosis on diroximel fumarate achieved no evidence of disease activity-3 status vs younger patients.

The decision was based on findings from the phase 3 confirmatory Clarity AD study, in which treatment with the agent resulted in significant reductions in amyloid-ß in the brain.

Patients on Hyqvia experienced longer time to relapse compared with those on placebo, with Kaplan-Meier estimate curves separating early, at approximately week 4.

METEOROID is the first study to evaluate the efficacy and safety of satralizumab, an FDA-approved therapy for NMOSD, in patients with MOGAD.

At 1 year, treatment with the investigational cell therapy was well tolerated with no major safety issues and showed evidence of cell survival and engraftment.

The professor of anesthesiology at Washington University in St. Louis discussed the importance of effectively communicating and reciprocating with underserved communities that are of research interest.

Among sleeping pill nonusers, when compared with patients who got a medium amount of sleep, extremely short or long sleepers had approximately 30% higher risk of all-cause mortality and 3-5 years shorter life expectancy at age 30-50 years.

Reasons behind the CRL were not related to efficacy and do not impact Amneal’s 2023 financial guidance, which did not include IPX203 revenues.

Through a quantitative electroencephalogram, investigators observed brainwave changes in several regions of the brain following treatment with CT1812.

Mind Moments®, a podcast from NeurologyLive®, brings you exclusive interviews with Amit Bar-Or, MD; Gavin Giovannoni, MBBCh, PhD; Dayna Johnson, PhD, MPH, MSW, MS; John Winkelman, MD, PhD; Laxman Bahroo, DO; Ling Wan-Albert, OTD, OTR/L; Sara Pavitt, MD; and Ali Ezzati, MD. [LISTEN TIME: 28 minutes]

The professor of anesthesiology at Washington University in St. Louis provided insight on his presentation at AHS 2023 discussing the need to engage underserved populations.

With the approval, Roche has 3 FDA-cleared tests that are reflective of the main Alzheimer disease pathologies and help clinicians more completely define the disease biologically.

Marisa Whalen, PharmD, associate director of Medical Affairs at Jazz Pharmaceuticals, discussed presentations at SLEEP 2023 on the real-world burdens of idiopathic hypersomnia and the smooth transition to low sodium oxybate in patients with narcolepsy.

In a phase 3 study, rozanolixizumab demonstrated statistically significant differences vs placebo on primary and secondary outcomes over a 43-day treatment period.

At 6 months, a larger proportion of patients on standard of care experienced unfavorable outcomes, as demonstrated by modified Rankin Scale scores greater than 3, in comparison with lumbar drainage.

In addition to increased relapse rates, use of fingolimod was associated with a 49% higher risk for disability accumulation in comparison with ocrelizumab.

Following the positive results, uniQure expects to initiate a third cohort in the ongoing trial to further investigate both low and high doses of AMT-130 in combination with perioperative immunosuppression.

In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.

In a trial of 250 adults with sporadic ALS, pegcetacoplan failed to meet its primary end point of change in Combined Assessment of Function and Survival rank scores relative to placebo after a 52-week stretch.

High and low doses of frexalimab resulted in reductions of new or enlarging T2 lesions and total T1 gadolinium-enhancing lesions over a 3-month period.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Natalie Goedeker, CPNP. [LISTEN TIME: 12 minutes]

Compared with placebo, inebilizumab-treated participants showed attenuated biomarker elevation during attacks and reduced biomarker levels over time in the absence of adjudicated attacks.