Neuromuscular Disease
FDA approves Soliris for patients aged 6 years and up with generalized myasthenia gravis
The FDA has approved an expanded indication of Soliris, an IV-administered monoclonal antibody for patients aged 6 years and older with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive.
Prenatal air pollution exposure tied to increased risk for cerebral palsy
Prenatal exposure to ambient air pollution was associated with increased risk for children developing cerebral palsy, according to cohort study results published in JAMA Network Open.
Radicava ORS demonstrates long-term safety, tolerability in patients with 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.
FDA clears electrical muscle stimulator suit for neuromuscular injuries, disease
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.
Amylyx announces end of enrollment for phase 3 trial of ALS therapeutic
Amylyx Pharmaceuticals Inc. announced completion of enrollment for a global, phase 3 clinical trial of AMX0035, an oral, fixed-dose medication for ALS.
TDP-43 accumulation likely indicator for patients with ALS
Presence of axonal phosphorylated transactive response DNA-binding protein 43, or pTDP-43, accumulation in intramuscular nerve bundles is a likely biomarker for patients diagnosed with ALS, according to a study published in JAMA Neurology.
High mortality, health care use for patients with hypercapnic respiratory failure
Hospitalized patients with compensated hypercapnic respiratory failure have high rates of mortality and health care utilization, with higher elevated carbon dioxide partial pressure associated with worse survival, researchers reported.
Among activity types, moderate exercise provides best ALS protection
Lifetime physical activity did not appear to increase risk for ALS, whereas occupational physical activity seemed to be a risk factor for the disease, according to study results published in Neurology.
Vamorolone demonstrates efficacy, safety for boys with Duchenne muscular dystrophy
Treatment with vamorolone, an anti-inflammatory steroidal drug, improved motor outcomes in boys with Duchenne muscular dystrophy compared with corticosteroid-naive individuals, according to findings published in PLoS Medicine.