Marco Meglio

Although a small sample of 5 patients with DMD, results showed significant expression of microdystrophin and reductions in creatine kinase, a biomarker of muscular distress, in GTN0004-treated patients.

Deflazacort, a corticosteroid approved for children with Duchenne muscular dystrophy, has been on the market since 2017 under the name Emflaza.

John Novak, MD, MS, director of the OhioHealth ALS Clinic and vice chair of the ALS Association Care Services Committee, provided insight on the upcoming ALS Nexus Conference, the first ever educational event from the organization, spanning multiple days in July.

The FIREFISH study showed that most children maintained or improved motor functions and feeding abilities over five years.

In a pivotal, large-scale, phase 3 trial, donanemab met its primary end point, demonstrating more pronounced effects in patients with early-stage Alzheimer disease who had low/medium tau status.

The Minority Stroke Program, open since 2019, encompasses a group of highly trained stroke experts who provide high quality care and education for minority ethnic patients presenting with stroke.

If the sBLA is approved, the clinical and biomarker benefits may be maintained through the once-monthly dosing regimen that is less burdensome and easier for patients and care partners to continue long-term.

After 28 days of treatment, bezisterim showed pronounced effects on measures of the Non-Motor Symptom Scale, as well as greater achievement of ON time than levodopa alone.

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 stroke and large hemispheric infarction.

In a subgroup of patients who underwent endovascular thrombectomy in addition to IV glibenclamide, results showed improved functional outcomes favoring active treatment, coupled with greater reductions in mortality.

Biogen's tofersen, an antisense oligonucleotide, remains the first and only marketed therapy to treat patients with SOD1-mutated amyotrophic lateral sclerosis.

Daridorexant, an FDA-approved dual orexin receptor antagonist, had a dose-response relationship observed at month 1 on all 4 efficacy end points for all doses tested.

The pulmonologist at Rush Health and immediate past president of the American Academy of Sleep Medicine provided perspective on some of the intriguing changes to sleep medicine and where the field is headed in the coming years.

Assessing doses of 3 mg doxepin, results revealed non-statistically significant improvements in latency to sleep onset and latency to persistent sleep that were observed across patients above and at or below the median baseline values.

A phase 3 trial plans to investigate the efficacy and safety of AD109, a novel oral medication, for treating obstructive sleep apnea in patients who cannot tolerate continuous positive airway pressure therapy.

Regardless of whether patients were treatment-naïve or taking alerting agents, the safety profile of low-sodium oxybate remained true, with treatment-emergent adverse events attributing to less than 4% of study discontinuations.

Overall, most patients reported satisfaction with cognitive behavioral therapy approach, with completion of lessons associated with a decrease in Fatigue Severity Scale.

Those on low-sodium oxybate, regardless of psychiatric comorbidity status, outperformed placebo on a number of outcomes, including Epworth Sleepiness Scale and weekly cataplexy attacks.

A majority of the small cohort of patients with SMA did not need a polysomnogram at 1 year because respiratory pathology was not a concern, attributable to improvements from disease-modifying therapies.

After 5 weeks of open-label treatment, KP1077-treated patients showed mild adverse events that were typical for a central nervous system stimulant.

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 amyotrophic lateral sclerosis (ALS).

Study establishes protocols for ublituximab use in breastfeeding patients with MS, enhancing treatment decision-making.

The phase 3 trials are expected to assess the therapeutic potential of frexalimab dosed every 4 weeks in both patients with relapsing MS and non-relapsing secondary progressive MS.

In comparison with adults on ocrelizumab, pediatric patients experienced no clinical relapses and a safety profile that was similar to what was previously observed.

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Katherine Peters, PhD. [LISTEN TIME: 13 minutes]

Within a subgroup of those older than 50 years, most patients who switched to cladribine were relapse free at follow-up, with no new safety signals observed.

Mike Hooven, chairman and CEO of Enable Injections, and Randall Moreadith, MD, PhD, chief development officer of Serina Therapeutics, provided insight on the development plans of SER-252, an investigational apomorphine therapy for advanced Parkinson disease.

The newly added tablet strengths allow for greater flexibility when selecting or adjusting dosing for deutetrabenazine.

Over a 24-month treatment period, patients on cladribine demonstrated no axonal loss evident on OCT, with reductions in cerebrospinal fluid neurofilament light and impacts on oligoclonal bands.

Nearly half of patients on diphenhydramine, dexamethasone, and famotidine experienced moderate to severe decreased alertness vs only 12.9% of those on cetirizine, dexamethasone, and famotidine.