Matt Hoffman

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.

After showing significant benefits in delayed disease progression in patients with secondary progressive MS, new data suggests siponimod can delay the time to wheelchair dependence by 4 years.

The professor of chemistry and chemical biology at Harvard University and Blavatnik National Laureate discussed the work she and colleagues have done on microbiome metabolism and what upcoming plans to pursue this further could achieve.

Year-long data presented at World Sleep 2019 suggest that Eisai’s lemborexant is safe and effective in treating adults with insomnia, supporting its recently submitted NDA. It is set to be reviewed by the FDA by late December.

Data presented at World Sleep 2019 has suggested that the investigation Jazz Pharmaceuticals sodium oxybate agent, JZP-258, is efficacious and safe in treating patients with narcolepsy who have cataplexy.

This regulatory approval in the treatment of pediatric patients aged 2 years and older with upper limb spasticity, excluding those with cerebral palsy, marks abobotulinumtoxinA (Dysport, Ipsen) as the first botulinum toxin approved for both upper and lower pediatric spasticity.

Following the receipt of a refusal to file letter from the FDA, Zogenix has resubmitted its application for fenfluramine hydrochloride (Fintepla), seeking approval for the treatment of seizures associated with Dravet syndrome.

The clinical development of ND0612 includes 2 doses; a low dose for mid-stage Parkinson patients experiencing loss of motor control on oral therapy, and a high dose for severe disease in which oral drugs are no longer effective.

Acorda Therapeutics’ Inbrija, an inhalation powder formulation of levodopa, was associated with no significant increases in troublesome dyskinesia for patients being treated with carbidopa/levodopa.

Data of amantadine (Gocovri, Adamas) extended-release tablets suggested that the therapy is, in fact, safe and tolerable with a unique pharmacokinetic profile, and results in significant improvements in dyskinesia for patients with Parkinson disease.

Acadia Pharmaceuticals’ pimavanserin significantly improved Hamilton Depression Rating Scale scores for patients with Parkinson disease with depressive symptoms in an 8-week, open-label study.

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.

PML incidence in a cohort of more than 6000 patients have decreased by 23% each year since risk guidelines and new serologic testing were introduced.

Based on datasets from 2 national registries and the European Interferon Beta Pregnancy Registry, the EMA’s Committee for Medicinal Products for Human Use has given a positive opinion to remove the contraindications, which would allow the Biogen agents to be used by pregnant and breastfeeding women with multiple sclerosis.

The analyses focus on the abuse potential, efficacy in high-burden patients with narcolepsy, and safety data for Harmony Biosciences’ histamine H3 receptor-antagonist/inverse agonist, pitolisant.

Analysis suggests that high-intensity aerobic exercise intervention, when gamified and delivered in an at-home fashion, can attenuate the symptoms of Parkinson disease while maintaining good adherence.

Data to be presented at the 2019 European Pediatric Neurology Society Congress suggest Zolgensma (onasemnogene abeparvovec-xioi) is associated with major milestone gains in SMA type 1.

A prospective, cross-sectional assessment suggests that patients with DMD can be adequately assessed for cognition in a brief period of time using the NIHTB‐CB, and additionally suggested a cognitive vulnerability in mothers who were carriers of DMD mutation.

Further analysis of the SHINE trial suggest nusinersen is linked to 6-year maintained improvement in patients with SMA. Biogen has announced a new global trial of a 50 mg loading dose in a broader cohort.

In a time where more than half of all patients with acute stroke and most patients with severe stroke arrive at the hospital via emergency medical services, a study suggests mobile stroke units are better equipped to accurately triage patients.

Mallinckrodt’s repository corticotropin injection was shown to have a lower cost per patient response compared to other late-line treatments for multiple sclerosis relapses, costing an estimated $148,528 less per response.

The GeNeuro agent showed marked reductions in brain atrophy in the thalamus and cerebral cortex through a long-term extension period treating patients with multiple sclerosis.

Impel NeuroPharma's nasal dihydroergotamine mesylate acute migraine therapy INP104 has shown excellent pharmacokinetic data, and is set to be assessed in a 24-week trial with a planned 28-week extension.

The director of the Sagol Center for Hyperbaric Medicine and Research spoke about how hyperbaric oxygen therapy may offer new hope in improving cognition in patients who have had a stroke.