SOD1

Tofersen, an experimental treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, failed to significantly slow the rate of disease progression relative to a placebo in a Phase 3 trial. These top-line findings mean that the VALOR study (NCT02623699), which is testing tofersen in…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. A new clinical trial will seek to determine the optimal timing to begin treatment with the investigational medication tofersen in people…

For the first time, a compound was able to restore the health of diseased upper motor neurons — nerve cells that carry voluntary movement signals from the brain to the spinal cord — in the brain of amyotrophic lateral sclerosis (ALS) mouse models, a study…

Newly developed compounds derived from ebselen could be used to treat some types of amyotrophic lateral sclerosis (ALS), a recent study suggests. The study, “Novel Selenium-based compounds with therapeutic potential for SOD1-linked amyotrophic lateral sclerosis,” was published in EBioMedicine. About a fifth of familial…

A team at the Rowan University School of Osteopathic Medicine has received a $1.6 million grant to study the role of the SOD1 protein — whose gene is often mutated in amyotrophic lateral sclerosis (ALS) — in the communication between the gut and the brain. The National Institutes…

A $25,000 Ionis Pharmaceuticals scholarship will help amyotrophic lateral sclerosis (ALS) patients in the San Diego, California, area participate in a host of integrative physical and mental wellness programs through Adapt Functional Movement Center. The full range of personalized sessions will be provided online during the COVID-19 pandemic. Designed…

A molecule that prevents cell death was able to prevent and reverse clumping of a mutant form of the protein superoxide dismutase 1 (SOD1) — the underlying cause of some familial amyotrophic lateral sclerosis (ALS) cases.  …

A single protein called Gemin3 may be the bridge linking the varied proteins known to underlie amyotrophic lateral sclerosis (ALS), suggesting a common pathway in this disease, a study using a fruit fly model showed.  The discovery of this common…

A toxin produced by algae may contribute to the development of amyotrophic lateral sclerosis (ALS) by lowering the stability of a protein in the brain, a new study suggests. Titled “β-Methylamino-L-alanine substitution of serine in SOD1 suggests a direct role in ALS etiology,” the study was…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…