Matt Hoffman

The FDA has granted the go-ahead to Biogen and Alkermes’ diroximel fumarate, which will be marketed as Vumerity, for the treatment of relapsing-remitting and secondary progressive MS, as well as clinically isolated syndrome.

Data presented at the Child Neurology Society Annual Meeting suggest that the safety and efficacy of cannabidiol (Epidiolex; GW Pharmaceuticals) is maintained through 72 weeks of treatment for patients with LGS.

VTS-72 is a proprietary combination of fumarate and VTS-Aspirin believed to improve the pharmacokinetics of fumaric acid while improving flush, which is estimated to occur in up to 40% of patients on a fumarate agent.

The agency sent a joint letter with the FTC to Rooted Apothecary LLC for claiming its CBD products can medically treat a number of conditions including Alzheimer disease and Parkinson disease. Since 2017, the FDA has issued more than 10 such letters.

SynapCell and Motac Neuroscience recently announced a novel biomarker, BetaPark [evo], for Parkinson disease progression. The chief innovation officer at SynapCell provided insight into the clinical implications of this discovery.

The director of the Parkinson’s Disease and Movement Disorders Center of Boca Raton and principal investigator of the PROSPECT study, offered insight into the clinical impact and advantages of the Cala Trio neuromodulation device.

The wrist-worn wearable neuromodulation device for essential tremor was shown to be safe and efficacious in the PROSPECT trial, with more than 50% of patients experiencing a ≥50% improvement in tremor "power" or severity.

The VMAT2 inhibitor marketed as Austedo by Teva Pharmaceuticals was associated with clinically meaningful long-term treatment benefit in patients with tardive dyskinesia, showing higher response rates than those observed in short-term trials.

New study results suggest that a wearable ECG device can predict nonconvulsive seizures with a sensitivity of 93.1% and all seizures with a sensitivity of 90.5%.

The director of pediatric epilepsy at Northeast Regional Epilepsy Group and co-director of epileptology at Hackensack University Medical Center provided context to these findings to explain how soticlestat can address challenges in developmental epileptic encephalopathies, as well as what may be next in its advancement.

Treatment with the Catabasis Pharmaceuticals small molecule oral NF-kB inhibitor resulted in improvements in the 10-meter walk/run scores, time-to-stand scores, and 4-stair climb scores in 31 steroid-naïve boys with DMD aged 4 to 7 years.

The director of pediatric epilepsy at Northeast Regional Epilepsy Group and co-director of epileptology at Hackensack University Medical Center spoke about soticlestat and what can be gleaned from data thus far.

Recent study results suggest that relative seizure reduction is an equivalent predictor of post-surgery quality of life in epilepsy to seizure freedom. Study author Lara Jehi, MD, offered insight into the value of these data.

A first-of-its-kind study has explored the social and professional impacts that tardive dyskinesia-related movements can have on individuals with the condition, as well outward perceptions.

Relative seizure reduction was as equally strong a predictor of patient quality of life after epilepsy surgery as seizure freedom in a recent study, with absolute reduction proving to be the worst model surveyed.

New study results suggest that thrombectomy may have similar rates of favorable neurologic outcomes and a comparable safety profile in both pediatric and adult patients with stroke.

A composite score of a number of measurements of evoked potentials may be able to predict sustained accumulation of disability in patients with clinically isolated syndrome, which could provide clinicians insight into early disease.

Alder Biopharmaceuticals announced the dosing of the first patient with ALD1910, its investigational monoclonal antibody that inhibits PACAP to prevent migraine. The study is aiming to enroll 100 individuals with results in mid-to-late 2020.

Eli Lilly’s lasmiditan (Reyvow) tablets are the first member of the ditan class approved for use in adults with migraine with or without aura, offering patients who have an inadequate response to other therapies a new acute treatment option.

The study unearthed a 20% diagnostic error rate with non-sex-specific parameters—10% false negatives in women and 10% false positives in men—which the authors found may be eliminated with gender-specific norms and cut scores.

This is the first study to compare infection risk with rituximab alongside other highly effective disease-modifying therapies.

The impact MT1621 had on survival probably for those treated in comparison to the natural history of the often-fatal disorder was highly significant, with all patients remaining alive as of October.

The senior director of Global Clinical Science at Takeda Pharmaceutical offered perspective on the development of TAK-925, an investigational selective orexin type-2 receptor agonist for the treatment of narcolepsy.

Treatment with the Catabasis Pharmaceuticals small molecule oral NF-kB inhibitor resulted in improvements in the 10-meter walk/run scores, time-to-stand scores, and 4-stair climb scores in 31 steroid-naïve boys with DMD aged 4 to 7 years.

New 9-month data suggest that Sarepta Therapeutics’ investigational limb-girdle muscular dystrophy type 2E treatment, SRP-9003, is linked to improved functional measures and reduction in creatine kinase.

As vestibular migraine is a common cause of vertigo, the results, which need to be confirmed with larger studies, suggest some potential for an effective and easy to use treatment for patients.

Cerevel Therapeutics’ highly selective dopamine D1/D5 agonist showed significant reductions in UPDRS-III scores for those with early-stage Parkinson disease and is planned to enter phase 3 in 2020.

Mallinckrodt’s repository corticotropin injection (Acthar Gel) showed statistically significant results for every efficacy end point assessed, including the primary end point. Data will be shared at an upcoming medical meeting.

Originally approved by the FDA in January, sumatriptan nasal spray was acquired by Upsher-Smith in a territory rights agreement with Dr. Reddy’s Laboratories in June, as well as its 3-mg injection formulation (Zembrace SymTouch).

The chief medical officer at Harmony Biosciences discussed the takeaways from a number of data presentations of pitolisant’s efficacy and safety from World Sleep 2019.