Patient enrollment is complete in a Phase 2b/3 clinical trial evaluating MN-166 (ibudilast), an investigational oral therapy developed by Medicinova to treat amyotrophic lateral sclerosis (ALS). The COMBAT-ALS (NCT04057898) trial is testing whether MN-166 can safely slow ALS progression in 234 adults with ALS, ages 18 to…
Long-read DNA sequencing is a more accurate method than short-read sequencing for detecting certain defects in genes associated with an increased risk of sporadic amyotrophic lateral sclerosis (ALS), a new study suggests. A relatively new technique, long-read sequencing can capture data on thousands to hundreds of thousands of nucleotides,…
AP-101, AL-S Pharma’s amyotrophic lateral sclerosis (ALS) therapy, was safe and well tolerated and led to clinically meaningful benefits for breathing and survival after one year of treatment. That’s according to topline data from a now-complete Phase 2a study (NCT05039099) that tested the therapy in 73 adults with…
China’s National Medical Products Administration has granted Sineugene Therapeutics permission to begin clinical trials of its gene therapy SNUG01 in people with amyotrophic lateral sclerosis (ALS), clearing the way for clinical trials. The announcement follows a similar decision by the U.S. Food and Drug Administration (FDA). It sets…
Long-term exposure to sulfur dioxide (SO2), a gaseous air pollutant generated by the burning of fossil fuels, significantly increases the risk of developing amyotrophic lateral sclerosis (ALS), according to a study in Canada. Exposure to other forms of air pollution, including nitrogen dioxide (NO2), ground-level ozone (O3), and PM2.5…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sineugene Therapeutics‘ SNUG01, a gene therapy for people with amyotrophic lateral sclerosis (ALS). The designation is given to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…
Living close to water bodies with blue-green algae blooms, or cyanobacteria, is linked to significantly reduced survival among people with amyotrophic lateral sclerosis (ALS), according to a new study. The association was significantly stronger in people who used private well water or participated in water-related activities such as swimming…
Researchers have discovered a pathway in cells that prevents the formation of TDP-43 protein clumps, which contribute to nerve cell damage in nearly all people with amyotrophic lateral sclerosis (ALS). The findings may aid ALS therapies that can activate the pathway and potentially limit TDP-43 clumping and prevent nerve…
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…
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…
