Matt Hoffman

Despite the clinical benefit being limited, the slowing of pantothenate kinase-associated neurodegeneration disease progression—particularly in atypical cases—is a step toward developing a treatment for the orphan disease.

During the average follow-up period of 3 years, 70.7% of those in the pediatric-onset MS group met the clinical definition for cognitive impairment compared with 59.8% of those in the adult-onset group.

The systematic review and meta-analysis identified cNfL usefulness as a biomarker, with the potential to help differentiate between frontotemporal dementia and Alzheimer disease, as well as Parkinson disease from atypical parkinsonian syndromes.

Patients with multiple sclerosis who were obese had higher EDSS scores and higher levels of inflammatory IL-6 and leptin in the CSF prior to treatment, as well as lower levels of anti-inflammatory IL-13.

Axovant reported positive safety data as well as improvements in a number of measurements of motor function and dyskinesias in patients with Parkinson disease who received treatment with their investigational gene therapy.

Almost 25% more of the patients with focal drug-resistant epilepsy who successfully underwent cortical stimulation to induce electroclinical seizures experienced good outcomes versus poor, suggesting the method’s ability to identify the epileptic generator as reliably as spontaneous seizures.

All told, every patient with RLS in the study had SIBO present, in comparison to general population rates which range from roughly 6% to greater than 15%.

Investigators noted that, when considered with efficacy findings in narcolepsy, pitolisant has a favorable risk-benefit profile representative of advancement in the treatment of excessive daytime sleepiness and cataplexy in adult patients.

The investigational dual-orexin antagonist from Eisai was significantly better at reducing Insomnia Severity Index-measured symptom severity in more than 1000 adults with insomnia in 2 phase 3 studies.

The Task Force sought to improve the employment of MRI’s capabilities in the treatment of epilepsy, which the authors noted “are variable worldwide and may not harness the full potential of recent technological advances for the benefit of people with epilepsy.”

The efficacy of CBT using the American Academy of Sleep Medicine (AASM) SleepTM platform has been shown to be similarly efficacious to face-to-face physician encounters in 30 adults with chronic insomnia.

The highly specific myostatin activation inhibitor is expected to enter phase 2 study in spinal muscular atrophy, according to SRK-015’s manufacturer, Scholar Rock.

Zilucoplan, also being developed for generalized myasthenia gravis, is expected to enter into a phase 2 clinical trial in IMNM in the second half of 2019, according to Ra Pharma.

New case-control findings suggest that glutamate and glutamine content may play a crucial role in the pathophysiology of functional motor symptoms.

Galcanezumab, an anti-CGRP antibody, is now the first approved therapy for the treatment of episodic cluster headache just 9 months after receiving approval for the preventive treatment of migraine.

The FDA designation was granted to the SK channel modulator after it showed promise in preclinical and phase 1 studies. Cadent Therapeutics is planning a phase 2a trial for the latter half of 2019.

The findings from an analysis of more than 2000 plasma samples suggest that levels of low-density lipoprotein cholesterol, independent of APOE, are associated with early-onset Alzheimer disease—and that the APOB gene may play a role.

Patients with favorable prognostic profiles or relapsing-remitting MS are the least likely to initiate high-efficacy disease-modifying therapies recommended by their physician, with many patients citing access challenges.

Results of a pooled analysis of 3 clinical trials suggest that the magnitude of fingolimod’s treatment effect in MS is not affected by the presence of migraine or headache in patients.

Data from 5 clinical trials and their extensions along with 1 real-world study suggest that treatment with teriflunomide can help patients of varying ages maintain low annualized relapse rates and stable EDSS scores.

Follow-up data extending out to 8 years suggest that both clinical and MRI outcomes are better with alemtuzumab (Lemtrada) than interferon beta-1a in patients with relapsing-remitting multiple sclerosis.

In those with MS treated with subcutaneous interferon beta-1a, Magnetic Resonance Imaging in multiple sclerosis scores may be predictive of the time to new clinical disease activity and disease progression.

The anti-CGRP agent was shown to decrease monthly migraine days, in addition to having higher ≥50% and ≥75% responder rates, reductions in acute medication use, and enhancements in MSQ RF-R domain scores for patients who failed ≥2 more preventives.

Despite a 40-fold increase in the use of LAAC in a 9-year period, those who had a history of stroke with major bleeds and higher comorbidity were less likely to have LAAC.

An MDA panel has stated that accessibility of advanced screening, the introduction of effective treatment, and the support of professional organizations could, and should, prompt the expansion of newborn screening.

The research fellow in the Department of Radiology at Mayo Clinic Jacksonville spoke about the reasoning for conducting this study and what the findings mean for physicians in clinical practice.

The automated system detected epileptic seizures and psychogenic non‐epileptic seizures with a sensitivity of 72.7% and specificity of 100%. The positive and negative predictive values for PNES classification were 81.3% and 100%, respectively.

The investigators suggested that this may be indicative of intrathecal IgG synthesis as a useful marker of disability worsening in patients with MS, as well as in making early treatment decisions.

Ultimately, the risk of peripheral neuropathy increased by an estimated 3% for each additional day of current fluoroquinolone exposure, maintained for up to 180 days post-exposure.

New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.