Neuromuscular Disease News

Rozanolixizumab improved myasthenia gravis symptoms in people with muscle-specific kinase antibody-positive disease, according to a poster presented at the 2023 MDA Clinical & Scientific Conference in Dallas.

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously that tofersen, an antisense oligonucleotide for the treatment of superoxide dismutase 1 ALS, may be considered for potential accelerated approval.

Researchers reported no differences in functional status or survival in patients with ALS treated with ravulizumab compared with placebo, according to a poster presented at the 2023 MDA Clinical & Scientific Conference.

Ravulizumab was associated with fewer clinical deterioration events in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis, according to a poster at the 2023 MDA Clinical & Scientific Conference.

Treatment with givinostat was associated with improvement in mobility and physical function compared with placebo in ambulant boys with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.

In a ceremony Monday at its annual meeting, the Muscular Dystrophy Association presented the 2023 Legacy Award for Achievement in Clinical Research to Merit Cudkowicz, MD, MSC, a renowned neurologist and researcher in ALS.

Results from a phase 3 clinical trial showed that Radicava ORS was safe in and well-tolerated by patients with ALS after 48 weeks of treatment, according to a manufacturer press release.

The FDA has approved Daybue for the treatment of Rett syndrome in patients aged 2 years and older, making it the first and only approved treatment for the genetic disorder, according to an Acadia Pharmaceuticals press release.

A new machine learning model that incorporates cardiac activity and body movement may help estimate the severity of Rett syndrome symptoms, enabling researchers to identify effective treatments, according to findings published in PLOS ONE.

The FDA has granted 510(k) clearance to Neuro20 Technologies for its Neuro20 Pro System, a wearable whole-body electrical muscle stimulator suit, software and operating system for neuromuscular injuries and disease.