Neuromuscular Disease News

Treatment with IV edaravone for ALS was linked to a slowing of disease-progression milestones compared with no IV edaravone treatment, results of an administrative claims analysis show.

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully human monoclonal antibody to treat individuals with spinal muscular atrophy.

A new analysis of an overall survival study of an investigational immunotherapy platform to treat ALS found it slowed disease progression and extended survival, according to the manufacturer.

The FDA has advised health care professionals and patients with multiple sclerosis that treatment with glatiramer acetate is associated with a risk for anaphylaxis and issued a new boxed warning, according to a drug safety communication.

The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic dystrophy type 1, or DM1, according to the manufacturer.

Treatment with tofersen in patients with superoxide 1 dismutase 1 ALS was linked to preservation of motor function, maintenance of muscle strength and reduction of disease-specific biomarkers, data show.

The FDA has granted priority review to a Biologics License Application for nipocalimab to treat individuals with generalized myasthenia gravis who are antibody positive, according to the manufacturer.

The average wait time between a referral and neurologist visit was more than a month among older adults, with longer wait times for those with serious neurodegenerative conditions like MS, epilepsy and Parkinson’s disease, new research shows.

Topline results from a phase 2/3 clinical trial showed that an eIF2B agonist to treat ALS failed to meet its primary endpoint of slowing disease progression compared with placebo at week 24.