News

Wave Life Sciences has gained additional financial support for an ongoing “basket” Phase 1b/2a clinical trial evaluating its experimental therapy, WVE-004, in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene. So-called basket trials, commonly used in the field of cancer, test…

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…

AI Therapeutics has launched a Phase 2 clinical trial into AIT-101 (apilimod dimesylate) as a potential oral treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations. The trial (NCT05163886) is currently recruiting at the Massachusetts General Hospital and is expected to soon open at sites in…

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…

Ady Barkan was a new father and activist when, at age 32, he learned that he had amyotrophic lateral sclerosis (ALS). His ensuing fight for universal healthcare while dealing with the progressive neurodegenerative disease is at the center of  “Not Going Quietly,” a film debuting on PBS today,…

Amarna Therapeutics and Redoxis have received an €800,000 (about $900,000) grant from Eureka Eurostars to advance their joint project, ALPHAVAC, which aims to discover immune-related therapeutic targets of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Promising targets, initially focused on ALS, then will be used to…

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…

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…