An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…

During my professional years working in a hospital environment, we had to be familiar with using medical jargon. Some words sounded odd to my nonmedical ears, while others had double meanings. A few favorites I still remember are “idiopathic,” which refers to something with an unknown cause, and “unremarkable,” which…

A mouse model of amyotrophic lateral sclerosis (ALS) carrying a mutation in the SOD1 gene showed alterations in the gut microbiome, followed by motor impairments and defects in enteric nervous system — the gut’s own autonomous nervous system — compared with healthy mice, a study found. These microbiome changes occurred…

Eikonoklastes Therapeutics has added a candidate gene therapy for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases to its pipeline. The announcement follows the completion of a licensing agreement with the University of California San Diego. Brian Head, PhD, a professor with the university’s department of anesthesiology, and…

Amyotrophic lateral sclerosis (ALS) can be grouped into four disease subtypes based on patterns of changes in electrical signals in the brain, researchers have discovered. These findings may be valuable to predict future disability and life expectancy, and may help select the patients more likely to benefit in certain…

Note: This story was updated Jan. 25, 2022, to clarify that Target ALS has raised $90 million since its inception in 2010. Amyotrophic lateral sclerosis (ALS) has been a part of Daniel Doctoroff’s life for more than two decades. His father, Martin Doctoroff, died of the disease in 2002, and…

The French health authority ANSM has approved InFlectis BioScience’s request to launch a Phase 2 clinical trial testing its lead candidate IFB-088, in combination with riluzole, in people with bulbar-onset amyotrophic lateral sclerosis (ALS). The trial is expected to be conducted in France and Italy, and a similar request is being…

Urine levels of neopterin, a marker of inflammation, are linked to disease progression in people with amyotrophic lateral sclerosis (ALS), a study revealed. That means, according to researchers, that neopterin could be used as a biomarker to monitor disease status and determine whether certain treatments will be effective. The…

Most folks know me through my column, “Living Well With ALS,” and perhaps don’t know that for the past three years, I’ve also been a co-moderator of the ALS News Today Forums. I thoroughly enjoy this responsibility, and to bring a bit of levity to this online job,…

The U.S. Food and Drug Administration (FDA) has approved a Phase 2 clinical trial of the psychedelic ketamine, PharmaTher’s experimental treatment for amyotrophic lateral sclerosis (ALS). FDA approval was requested through an investigational new drug application (IND) submitted by the study’s principal investigator, Richard Barohn, MD, a neurologist…