An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels…
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Neurosense Therapeutics’ oral therapy PrimeC significantly reduces blood levels of miRNAs — molecules involved in regulating gene activity — associated with disease progression and survival in people with amyotrophic lateral sclerosis (ALS). That’s according to six-month biomarker data from the Phase 2b PARADIGM clinical trial (NCT05357950), in which…
Daily treatment with IPL344 for up to three years was safe and appeared to slow amyotrophic lateral sclerosis (ALS) progression, according to recently published data from a small Phase 2a clinical trial. Patients undergoing treatment gained weight and experienced slower declines in functional abilities and lung function. The…
The ALS Association is teaming with the workplace learning company Schoox to provide education resources for patients, caregivers, healthcare providers, and others affected by amyotrophic lateral sclerosis (ALS). With this partnership, Schoox will provide fast and easy access to a range of resources, including educational materials and…
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…
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…
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