Matt Hoffman

The International Headache Society has issued a number of recommendations for the proper design of trials for the prevention of pediatric migraine, hoping to address the challenges which are unique to pediatric patient populations.

Data from a national cohort has shown a 48% rate of confirmed symptomatic fractures, which the authors suggested was expected to be a large underestimation of the real frequency in the DMD patient population.

According to Kyowa Kirin, the investigational selective adenosine A 2A receptor antagonist has had its PDUFA action date set for August 27, 2019.

The MEK 1/2 inhibitor was previously granted an orphan drug designation by the FDA in February 2018 and resulted in partial responses in 72% of patients in the phase 2 SPRINT trial.

The professor of neurology at Harvard Medical School discussed differentiating pediatric MS from other CNS disorders, as well as the process of treating and managing these patients.

Experts say that while CBD and THC may have potential in multiple sclerosis and epilepsy, research on the endocannabinoid system is in its infancy—and the literature isn’t perfect.

The EMD Serono product was approved based on data which displayed a significant decrease in the number of relapses experienced by patients with MS who had ≥1 relapse in the previous year, compared to placebo.

The study author, a pediatric neurologist at Baylor College of Medicine and director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, spoke about the investigational Rett syndrome treatment’s potential going forward.

The neurologist at Cleveland Clinic’s Mellen Center provided his insight into the clinical evidence for the selective sphingosine 1-phosphate receptor modulator.

With the lack of a gold standard diagnostic test, a study has shown through latent class analysis that cerebrospinal fluid immunoglobulin G may be a practical tool for determining the onset of MS in patients, particularly those who tested negative under the 2005 and 2010 McDonald criteria.

Of the 5 syndrome-specific efficacy measurements, trofinetide showed statistical benefits for patients with Rett syndrome on the Rett Syndrome Behavior Questionnaire, Clinical Global Impression Scale-Improvement, and the RTT Clinician Domain Specific Concerns-Visual Analog Scale.

Topline data showed that the novel oxybate candidate demonstrated significant differences compared to placebo in the change in the weekly number of cataplexy attacks and the change in Epworth Sleepiness Scale score, the primary and secondary end points, respectively.

The novel longitudinal model utilized 4 variables—the presence of seizures, the number of seizures, the number of adverse events, and if treatment was altered—and was ultimately more predictive than a standard Cox model.

Marketed as Mayzent, the selective sphingosine 1-phosphate receptor modulator is the first treatment for patients with active secondary progressive multiple sclerosis in almost 15 years.

Celgene is supporting its new drug application for the S1P receptor modulator with data from the RADIANCE and SUNBEAM phase 3 trials in MS.

The Movement Disorder Society Task Force on Technology has released a roadmap for the implementation of new technologies that have reached a level of sophistication, versatility, and wearability for Parkinson disease.

Findings from a recent meta-analysis of the number of atypical antipsychotics available for treatment of behavioral and psychological symptoms of dementia suggest that a clear trade-off exists between effectiveness and safety, with no clear winner emerging.

SYROS study data showed that switching to and maintaining long-term treatment with idebenone was associated with a 50% reduction in the annual rate of decline in forced vital capacity percent of predicted.

With a pair of sBLAs under FDA review, the Allergan treatment may finally have an official indication for use in spasticity after years of off-label use by physicians.

Study results suggest that no difference in outcomes of function, survival, fatigue, or quality of life are seen between those undergoing intensive or standard exercise regimens.

Eisai and Biogen have announced that the phase 3 ENGAGE and EMERGE trials, in addition to the PRIME phase 1b and EVOLVE phase 2 studies, of the monoclonal antibody, will be discontinued.

The migraine specialist spoke to the CGRP receptor antagonist’s potential impact on patients with migraine who have been lacking a novel acute treatment for some time.

An analysis of the EARLYSTIM study data has suggested that the 39-item Parkinson’s Disease Questionnaire summary index may be predictive of the future outcomes of subthalamic deep brain stimulation.

The multiple sclerosis specialist at Baylor Scott & White Health spoke about which symptoms present the biggest challenges for providers.

The staff neurologist at Premier Neurology, a partner in care with the National MS Society, spoke about how to best overcome the challenges presented by the symptomatic management of MS.

The director of the Medstar Georgetown University Headache Center spoke about the data that’s been presented thus far, as well as how ubrogepant separates itself from what’s currently available for patients.

The neurologist with Premier Neurology spoke about the challenges and state of symptomatic care in multiple sclerosis.

After almost a year on the market, post-marketing data has been suggestive of a risk of anaphylaxis and angioedema in patients who are hypersensitive to erenumab or the excipients.

Ned Sharpless, MD, the director of the National Cancer Institute, has been named to the position of acting FDA Commissioner. The announcement was made just a week after the current commissioner, Scott Gottlieb, MD, announced his plans to resign in early April.

The 2-year safety and efficacy extension of the CHANGE-MS trial ultimately showed encouraging dose-dependent effects with the 18-mg/kg dose on confirmed 12-week EDSS and 25-foot timed walk scores.