Cognitive deficits associated with amyotrophic lateral sclerosis (ALS) are linked to a particular pattern of abnormal changes and altered blood flow in specific brain regions, a study shows. The study titled “Brain Structural and Perfusion Signature of Amyotrophic Lateral Sclerosis With Varying Levels of Cognitive Deficit” was…

The biotech company Kadimastem is recruiting for a Phase 1/2a clinical trial to test the safety and effectiveness of its leading cell therapy, called AstroRx, in patients with amyotrophic lateral sclerosis (ALS), after positive results were seen in animal models of the disease. The study showed that AstroRx, made…

Specific alterations in RNA processing are molecular hallmarks of familial and sporadic forms of amyotrophic lateral sclerosis (ALS), a new study suggests. This research might lead to more discoveries  about how ALS develops and ultimately contribute to a cure. The study, “Intron retention and nuclear loss of SFPQ are…

Results from a Phase 1 clinical trial reveal that giving patients infusions of a specialized immune cell may be a viable option to safely slow the progression of amyotrophic lateral sclerosis (ALS). That finding was reported in the study “Expanded autologous regulatory T-lymphocyte infusions in ALS,” published…

A high genetic risk for amyotrophic lateral sclerosis (ALS), assessed using so-called polygenic risk scores,  was linked in a study to poorer performances on verbal and numerical tests in otherwise healthy adults, but not to physical disabilities. The study, “Genetic risk for neurodegenerative disorders, and its overlap with cognitive…

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…