Neuromuscular Disease News

Viltepso, an injection treatment for Duchenne muscular dystrophy in patients with a confirmed mutation, demonstrated a statistically significant benefit for motor function in open-label extension results from a phase 2 study.

Exservan, the first oral film formulation of riluzole, has been made available in the United States for the treatment of ALS, according to a press release from Mitsubishi Tanabe Pharma America, the drug’s manufacturer.

A survey of community neurologists demonstrated four primary themes associated with the impact of the COVID-19 pandemic in this group, specifically highlighting “the unusual environment” in which these providers practice.

Efgartigimod, a human IgG1 antibody designed to decrease pathogenic IgG autoantibody levels, demonstrated efficacy and tolerability among patients with generalized myasthenia gravis, according to findings published in The Lancet Neurology.

An analysis of exome sequences in patients with ALS and healthy controls “strongly” indicated that variants in the TP73 gene are associated with risk for ALS, according to study results published in Neurology.

Progressive degeneration of the motor cortex correlated with more aggressive clinical presentations among patients with ALS, according to findings from a prospective, longitudinal, multicenter study published in Neurology.

An international team of researchers at the NIH and other institutions have identified a novel form of ALS in a small set of children, starting at approximately 4 years of age, linked to the SPTLC1 gene, according to a press release.

Ongoing follow-up in the START phase 1 study supported “the long-term favorable safety profile” of Zolgensma in children aged up to 6 years with spinal muscular atrophy type 1, according to findings published in JAMA Neurology.

The American Academy of Neurology released an update to their 2014 position statement on telehealth in which they reiterate their call for expanding its use.

Researchers observed a significant difference in the rate of complete stable remission among several surgical methods for thymectomy in myasthenia gravis, according to results of a meta-analysis published in Neurology.