Neuromuscular

By the end of the open-label extension, 15-17% of patients showed clinically meaningful first response with eculizumab treatment.

Real-world, observational cohort data suggest that Biogen’s nusinersen (Spinraza) is safe and effective in the treatment of spinal muscular atrophy.

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

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

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.

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.

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

Statistically significant associations between stress-related disorders were identified in those with Alzheimer disease, but not with Parkinson disease or amyotrophic lateral sclerosis.

The chief medical officer of Clene Nanomedicine detailed CNM-Au8, their novel investigational drug currently being assessed in multiple trials for patients with multiple sclerosis and other neurodegenerative diseases.

As neurologists and other specialists may increasingly be called upon to serve as general hospitalists in the wake of the COVID-19 pandemic, keeping health care workers healthy becomes paramount.

The spine surgeon in the Center for Spine Health at Cleveland Clinic spoke to the importance of improving patient-reported outcomes and ensuring consistent communication with patients undergoing spine surgery.

The chief executive officer of Clene Nanomedicine discussed CNM-Au8, their novel investigational drug that will be used in the world’s first platform trial of potential ALS treatments.

Gaboxadol is the first treatment in 50 years associated with positive change in outcomes in Angelman syndrome.

The president and founder of Cure Rare Disease discussed the company’s custom therapeutics for patients with rare diseases, including the use of CRISPR gene-editing technology to develop treatments for Duchenne muscular dystrophy.

Understanding the pathophysiology of myasthenia gravis is still being determined to this day, but the neonatal Fc receptor is emerging as a potential treatment.

The Healey ALS Platform Trial learns from successful cancer drug studies to identify and accelerate new treatments for the devastating disease.

Neurology News Network for the week ending February 22, 2020.