Verge Genomics has identified multiple digital clinical biomarkers that can be used to assess short-term disease progression in the pre-treatment run-in period of a Phase 1b trial testing VRG50635, its treatment candidate for amyotrophic lateral sclerosis (ALS), in patients. These digital biomarkers measure changes in key functions, such…
A rare mutation that leads to abnormally high activity of the CREB3 protein is associated with a reduced risk of developing amyotrophic lateral sclerosis (ALS) and people with ALS who carry this mutation progress significantly more slowly and live longer than those who don’t. That’s according to the study,…
A patient with late-stage amyotrophic lateral sclerosis (ALS) was treated with TRE-515, Trethera’s investigational therapy, under a U.S. Food and Drug Administration (FDA) expanded access program, the company has announced. The program enables patients with life-threatening conditions, including ALS, to receive experimental therapies outside clinical trials when no…
Trace Neuroscience plans to test genomic medicine that targets the UNC13A protein in people with amyotrophic lateral sclerosis (ALS) in a Phase 1/2 clinical trial that will be supported by artificial intelligence (AI)-powered tools from Unlearn. Unlearn’s Digital Twin Generator for ALS (ALS DTG) will be used…
Edaravone, an approved amyotrophic lateral sclerosis (ALS) therapy sold as Radicava and Radicava ORS, may exert neuroprotective effects by correcting a key molecular feature of ALS, a cell-based study shows. Specifically, edaravone was found to correct the abnormal localization of TDP-43, a protein that is involved in many…
Last week for spring break, I traveled with my son from snowy Michigan to sunny Florida. Siesta Key was just as beautiful as I remembered from 15 years ago when my husband, Todd, and I vacationed there with our two small children, along with Todd’s parents and my brother…
Taking statins to lower cholesterol does not affect survival outcomes for people with amyotrophic lateral sclerosis (ALS), researchers in Norway have found. In a study of ALS patients using national health registry data, the scientists compared the life expectancy of statin users and nonusers. According to the team, “statin use…
I realize that the words “goals,” “improvement,” and “ALS” don’t always appear in the same sentence, but for me, they do. That’s because, over the past few years, I’ve followed a strategy of setting personal fitness goals and using my ALS clinic appointments as the mile markers for those…
Nine patients with an ultra-rare form of amyotrophic lateral sclerosis (ALS) caused by mutations in the CHCHD10 gene were treated with an experimental antisense oligonucleotide (ASO) that’s designed to silence the disease-causing mutations. The treatment was developed as part of Silence ALS, an initiative that designs ASOs, that is,…
The U.S. Food and Drug Administration (FDA) has authorized Sineugene Therapeutics to begin a Phase 1/2a trial of its experimental gene therapy, SNUG01, in people with amyotrophic lateral sclerosis (ALS). The global trial will assess the treatment’s safety, tolerability, and preliminary efficacy through a dose escalation and expansion…
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