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What do you suspect is responsible for this patient’s progressively worsening cognitive symptoms?

NeurologyLive spoke with the director of the Cleveland Clinic Lou Ruvo Center for Brain Health to learn why, despite hundreds of failed trials and therapies, there is growing excitement for what's to come.

Infants with SMA—who have elevated pNF-H levels—experienced significant drops in pNF-H levels when treated with nusinersen (Spinraza, Biogen), suggestive of pNF-H’s potential as a biomarker of disease activity and treatment response.

The Assistant Professor at the University of Alabama School of Medicine spoke about the results of a recent cohort study that found that a shorter, yet deeper therapeutic coma may be safer and more effective than the currently recommended therapeutic coma duration for refractory status epilepticus.

The study provides class 2A and Level B evidence that DOAC therapy started in a median of 5 days following ischemic stroke in patients with atrial fibrillation is beneficial compared with VKA therapy.

A prespecified futility analysis of a phase 3 study revealed that the primary endpoint of mean change from baseline in the weekly average number of cluster headache attacks during the 4-week treatment period is unlikely to be met.

Alder stated that it expected to receive a PDUFA action date within 74 days of the submission for eptinezumab and is planning a commercial launch of the anti-CGRP agent by early 2020.

Research from the past decade suggests that sigma-1 receptors and their encoding gene, SIGMAR1, together act as a therapeutic target for patients with dementia.

The chief executive officer of Alder Biopharmaceuticals detailed where the intravenous anti-CGRP treatment will fit into the marketplace and how the migraine prevention therapy will make an impact in treating patients.

What's your diagnosis for a 45-year-old woman who experienced strange symptoms after taking an advanced yoga class?

The approval of nusinersen in 2016 to treat spinal muscular atrophy marked an important milestone in efforts to treat the disease, but even more breakthroughs could be coming soon.

A review of newly implemented primary end points—pain freedom and most bothersome symptom freedom at 2 hours—used in 8 clinical trials may help to guide future migraine researchers in the design of acute treatment trials.

Responses from 1300 physicians suggest that increasing patient load and adding ancillary services may be the key to increasing annual income.

Neurology News Network for the week of April 20, 2019.

The designation was granted by the FDA based on the results of the largest study ever conducted with monotherapy in NMOSD, which were favorable for the anti-CD19 antibody.

With 2 targeted treatments on the market, therapeutic development may now focus on early detection.

In addition to efficacy end points, the WEB system was markedly safer compared to current treatments, with no primary safety end points occurring between 30 days and 1 year.

Phase 2 trial results suggest the possibility of a treatment that may regenerate brain cells following TBI.

The phase 1 results support the initiation of the phase 2/3 clinical trial which is expected to initiate in July 2019.

It has been estimated that between 40% and 60% of patients with PD use at least one form of complementary medicine. In this podcast, neurologist Sarah Mulukutla, MD, MPH talks about CAM options for neurologists to discuss with their patients.

Data from the STR1VE trial have reinforced the findings from the phase 1 START trial, including inclinations of prolonged survival as well as milestone achievement never seen in the natural history of SMA.

Alder Biopharmaceuticals’ monoclonal antibody was recently submitted in a BLA to the FDA and is on pace to hit the newly crowded preventive migraine market in early 2020.

Chronic pain, sleep disorders, neuropathy, and anxiety were the most common conditions that showed improvement in the study.

MediciNova will be moving forward with a phase 2b/3 clinical trial of the ALS therapy, also known as MN-166, in 150 patients. If successful, its data will be used to support an NDA.

Patients with multiple sclerosis who were exposed to teriflunomide during pregnancy experienced similar risks of major birth defects and spontaneous abortions to the general population’s rates of 2% to 4% and 15% to 20%, respectively.

The scale includes 11 observable characteristics of CDM1 and was established through the merging of experiences and advice from patients, caregivers, therapeutic experts, and the FDA.

The Aquestive Therapeutics product, marketed as Exservan, was previously granted an orphan drug designation in January 2018. Its NDA is supported by a number of studies which sought to confirm its bioequivalence to its reference listed drug.

The study also added to a growing base of findings which suggest that patients who have failed preventive medications prior to trial participation have lower placebo responses, which could have implications for clinical trial design.

The senior preclinical and clinical imaging scientist at the National Institute of Neurological Disorders and Stroke spoke about the potential of the central vein sign to help improve the time to an accurate diagnosis in MS.

In an ongoing phase 2 study, 21 nonambulatory participants with Duchenne muscular dystrophy passed the 52-week treatment mark.