Two young companies, QurAlis and Kernal Biologics, are winners of the Amgen Golden Tickets for innovative bioscience projects — in this case, work that might lead to better treatments in amyotrophic lateral sclerosis (ALS) and cancer immunotherapy, Amgen and LabCentral announced. QurAlis is developing therapies for three different forms of…
The Wave Life Sciences therapy WVE-3972-01 reduced markers of the form of ALS that scientists believe stems from a gene mutation, studies show. Researchers said the potential treatment blocked faulty versions of messenger RNA associated with the production of abnormal C9orf72 protein. The RNA is an intermediary between the C9orf72 gene…
MediciNova announced successful top-line results from a Phase 2 clinical trial of MN-166 (ibudilast) as an adjunct therapy to Rilutek (riluzole) in patients with amyotrophic lateral sclerosis (ALS). The treatment was safe and well-tolerated – meeting the trial’s primary endpoint – and was found to be more effective at…
Ludwig Cancer Research scientist Don Cleveland has received a $3 million Breakthrough Prize for increasing science’s understanding of how an inherited form of amyotrophic lateral sclerosis develops. His work focused on glia, cells that support the body’s production of a protein called myelin, which protects nerve cells. Deterioration of myelin…
Scientists have long believed that ALS was confined to one area of the brain, but an Irish study shows it affects a number of regions. Researchers at Trinity College Dublin’s Academic Unit of Neurology used electroencephalography, or EEG, to record patients’ brain activity. They discovered that nerve cell communication was lower…
Amyotrophic lateral sclerosis (ALS) patients with locked-in syndrome reported having a satisfactory quality of life and low levels of depression, in spite of their near-complete disability, in a small but recent study. The results of the questionaire-based study were presented by its lead author, Dorothée Lulé, at the 28th…
BrainStorm Cell Therapeutics released new data on potential biomarkers from its Phase 2 trial investigating NurOwn, the company’s cell therapy for amyotrophic lateral sclerosis (ALS), at the International Symposium on ALS/MND meeting held in Boston last week. Both these findings and an ongoing Phase 3 trial, now recruiting patients in…
Pivotal Phase 3 clinical trial results presented at this year’s 28th International Symposium on ALS/MNDÂ reveal that Cytokinetics‘ investigational therapy tirasemtiv failed to provide a positive therapeutic impact on both muscle strength and slow vital capacity (SVC) in ALS patients. The results, which were presented by Jeremy Shefner, MD,…
The rate of ALS patients’ lung-function decline can predict their risk of breathing failure, their need for assisted ventilation, and their risk of death, according to Cytokinetics, a company that develops muscle-disease therapies. “These results suggest that respiratory function is an important prognostic indicator of clinical progression in people with…
Additional data on Radicava (edaravone) and the clinical trial that led to its approval to treat amyotrophic lateral sclerosis (ALS) will be presented in oral and poster presentations at the 28th International Symposium on ALS/MND that opens in Boston on Friday. Mitsubishi Tanabe Pharma America announced that its one oral…
