Steve Bryson, PhD, science writer —

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…

Differences in tear metabolites — the products of metabolism that are found in human tears — in people with amyotrophic lateral sclerosis (ALS) were able to distinguish bulbar-onset disease, in which symptoms initially affect the face and throat, from spinal-onset ALS, where the first symptoms usually are weakness in…

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…

A $22 million federal grant to the Mayo Clinic in Florida will fund a study providing access to the investigational therapy ibudilast (MN-166) for people with amyotrophic lateral sclerosis (ALS) across the U.S. The award, by the National Institute of Neurological Disorders and Stroke (NINDS), will support a…

A computer analysis using real-world data from an amyotrophic lateral sclerosis (ALS) clinic identified four groups of patients with distinct rates of disease progression, a new study by researchers in Portugal reported. The findings continue to demonstrate the differences in ALS presentation and progression seen among people with the…

A new genetic mutation that may cause amyotrophic lateral sclerosis (ALS) has been discovered in families living in La Rioja, a region in Spain, according to a study. The gene mutation, found in 10 ALS patients from seven different families, affected the ARPP21 gene, which encodes a protein that…

Io Therapeutics is planning to launch a Phase 2 trial into its investigational oral therapy IRX4204 in people with amyotrophic lateral sclerosis (ALS). The decision follows recent tests showing that the candidate treatment dampened neuroinflammation in an animal model of neuronal autoimmunity, or diseases marked by self-directed…

Repeat infusions of naive immune B-cells were safe and delayed disease onset, reduced disease severity, and extended survival in animal models of amyotrophic lateral sclerosis (ALS), a study found. The approach was also deemed safe in the treatment’s first in-human case study, in which it eased disease severity and…

The use of nonpharmacological treatment — interventions such as muscle exercise, aerobics, and strength training — did not significantly ease pain among people with amyotrophic lateral sclerosis (ALS), according to a review of published data from five clinical trials. The researchers noted, however, that while “pain in ALS patients…

COYA 302, Coya Therapeutics’ experimental immune-modulating combination therapy, safely slows disease progression and reduces levels of disease biomarkers in people with amyotrophic lateral sclerosis (ALS). That’s according to the now-published results from a small proof-of-concept Phase 1 clinical trial (NCT06307301) that tested the therapy in four ALS…