News

Highly targeted approach supports ALS gene therapy development

Researchers have developed a type of gene therapy that can rescue the function of the TDP-43 protein in diseased nerve cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases while leaving healthy cells untouched. The approach uses problematic sequences called cryptic exons that are only present in cells…

Neuvivo requests FDA approval of ALS treatment NP001

Neuvivo has filed an application with the U.S. Food and Drug Administration (FDA) requesting the approval of its investigational therapy NP001 (sodium chlorite) to treat amyotrophic lateral sclerosis (ALS). The new drug application comes on the heels of combined data from Phase 2a (NCT01281631) and Phase…

Immune proteins activation at ALS diagnosis linked to progression

Researchers detected changes in the production of several immune signaling proteins, called cytokines, in the cerebrospinal fluid (CSF) — the liquid that surrounds the brain and spinal cord — of people with amyotrophic lateral sclerosis (ALS) at diagnosis. These changes also correlated with certain clinical characteristics, and how quickly the…

Target ALS, Modality.AI partner to study AI in ALS

Modality.AI is teaming up with Target ALS on a clinical study of artificial intelligence (AI) as way to assess changes in speech and motor function in people with amyotrophic lateral sclerosis (ALS). The trial will harness a virtual, conversational AI system, called Tina, intended to detect even…