Lower-than-usual levels of a molecule called miR126-5p — a type of microRNA, with a role in producing proteins — led to neuron degeneration and disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), in part by working to raise levels of toxic molecules, a study reports. Its researchers…

An experimental oral formulation of edavorone for amyotrophic lateral sclerosis (ALS), called TW001, demonstrated promising pharmacological and safety data in a Phase 1 clinical trial, according to the biotech Treeway. The randomized trial compared TW001 to Radicava (Mitsubishi Tanabe Pharma America), an intravenous medication approved…

Researchers discovered that a small region of ataxin-2 — a protein known to be involved in amyotrophic lateral sclerosis (ALS) — is crucial for both long-term memory and the formation of toxic aggregates that lead to neurodegeneration. The study with that finding, “RNP-Granule Assembly via Ataxin-2 Disordered…

A gene therapy that might treat familial amyotrophic lateral sclerosis (ALS), an inherited form of the disease, was recently granted orphan drug status by the European Medicines Agency (EMA), an award that carries incentives to promote potential treatments for rare diseases. The investigative therapy is in preclinical development…

Clinigen Group recently partnered with Mitsubishi Tanabe Pharma Corporation to launch an early access program in Europe for Radicava (edaravone), an intravenous treatment for amyotrophic lateral sclerosis (ALS). The therapy is not yet approved in Europe, but the early access program will help make it available…

Voyager Therapeutics presented new data from a study in an animal model of  its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…

A new mouse model developed by researchers at Karolinska Institute in Sweden revealed a molecular mechanism that may contribute to the development of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) or multiple sclerosis. The mouse model was described in the study, “Fatal demyelinating disease…

The protein TRIF can prevent resident immune cells of the brain from activating abnormally and slow the progression of amyotrophic lateral sclerosis (ALS), researchers in Japan working in a mouse model of the disease report. Their work, “Innate immune adaptor TRIF deficiency accelerates disease progression of ALS mice with…

The distinct processes that drive FUS proteins to aggregate and clump in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) were detailed in a new report. The study, “Phase Separation of FUS Is Suppressed by Its Nuclear Import Receptor and Arginine Methylation,” appeared in the journal Cell. The…

Worldwide Clinical Trials and the healthcare technology company Datavant are partnering in an effort to improve trials in neurological diseases such as amyotrophic lateral sclerosis (ALS). With the aging of Western populations, neurodegenerative diseases such as ALS, Alzheimer’s, Parkinson’s and Huntington’s represent an increasingly high…