News

Preliminary data from the ASPIRO study of Audentes Therapeutics resamirigene bilparvovec gene therapy suggests it is safe with improvements in neuromuscular and respiratory function.

Pear Therapeutics’ Somryst intervention provides tailored neurobehavioral interventions intended to improve the symptoms of insomnia, via cognitive behavioral therapy and algorithm-driven sleep restriction.

Patients with spinal muscular atrophy maintained the ability to thrive and achieved motor milestones previously unseen in natural history studies.

Although the drug requires no CYP2C9 genotyping or first-dose observation, an up-titration scheme should be used due to the risk of transient cardiac conduction issues.

The novel oxybate agent is seeking an indication for the treatment of cataplexy and excessive daytime sleepiness in those 7 years and older with narcolepsy. The PDUFA date is July 21, 2020.

Catabasis’s small molecule NF-kB inhibitor showed promise in phase 2, and the phase 3 PolarisDMD study (NCT03703882) of edasalonexant is fully enrolled and expected to read out in late 2020.

In addition to no-charge testing, patients may have access to the Detect Muscular Dystrophy program, a counseling program designed to aid health decisions and follow-up care.

Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.

The director of the Multiple Sclerosis Program at Cleveland Clinic’s Lou Ruvo Center for Brain Health detailed what can currently be surmised about siponimod’s effect in treating patients with SPMS.

Data from the DELOS and SYROS studies of idebenone suggest that investigational DMD treatment has a treatment effect maintained for up to 6 years, which can reduce hospitalizations, bronchopulmonary adverse events, and systemic antibiotic use.

No unexpected safety signals were reported in the post hoc analysis.

After meeting co-primary end points of pain freedom and freedom from the most bothersome symptom, the acute migraine agent has been cleared for a phase 3 trial by the FDA.

The director of the Multiple Sclerosis Program at Cleveland Clinic’s Lou Ruvo Center for Brain Health discussed the findings of a subanalysis of the EXPAND study of siponimod in patients with secondary progressive multiple sclerosis.

Eligible patients with Duchenne muscular dystrophy may receive the drug while it is under priority review by the FDA.

Patients with multiple sclerosis who reported cannabis use noted that it was helpful in relieving pain, as well as other symptoms, such as sleep, depression, anxiety, and/or stress.

The director of the Sleep Disorders Center and vice chair of the department of neurology at the UCLA David Geffen School of Medicine detailed the comorbidities related to narcolepsy.

The risk of relapse was higher with treatment of mycophenolate mofetil in patients with neuromyelitis optica spectrum disorder and other first-line immunosuppressants, compared to treatment with rituximab.

The chief medical officer at SK Life Science discussed what the clinical community needs to know with cenobamate headed to the market in the second quarter of this year.