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…

Size matters when it comes to SOD1 protein aggregates found in the brains of patients with amyotrophic lateral sclerosis (ALS), according to researchers. They found that large SOD1 aggregates may help protect motor neurons from dying, while short SOD1 aggregates promote the death of motor nerve cells. Their findings support…

A single dose of an artificial microRNA — a tiny RNA molecule that is able to control the activity of certain genes — can lower the activity of SOD1, a gene commonly mutated in patients with familial amyotrophic lateral sclerosis (ALS), a proof-of-concept study shows. According to researchers,…

A new study entitled “Aggregation propensities of Superoxide Dismutase G93 hotspot mutants mirror Amyotrophic Lateral Sclerosis clinical phenotypes” identified a mechanism that leads to the aggregation of SOD protein mutant forms that are typically found in Amyotrophic lateral sclerosis (ALS) motor neurons. The study opens new therapeutic avenues…

The first patient has been dosed in a Phase 1/2 clinical trial of AMT-162, the one-time gene therapy in development by uniQure for treating amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, when the condition is thus known as SOD1-ALS. The open-label EPISOD1 clinical…

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…

RAG-17, an experimental treatment Ractigen Therapeutics is developing for people with amyotrophic lateral sclerosis (ALS) who carry a SOD1 mutation (SOD1-ALS), was well tolerated at multiple doses and led to profound changes in disease biomarkers, preliminary trial data showed. The data cover the first part of an ongoing…

Molecular changes linked to inflammation and how brain cells process proteins and genetic material may occur before the onset of amyotrophic lateral sclerosis (ALS) symptoms, a study in a mouse model of SOD1-related ALS suggests. The findings also point to changes in the hypothalamus, a brain region involved in…

Some people with amyotrophic lateral sclerosis (ALS) who received Qalsody (tofersen) in the VALOR trial experienced improvements in physical function, lung function, and muscle strength over about three years of treatment, a new analysis found. Gains were observed more often among those who started treatment in the main…

ISIS Pharmaceuticals, in collaboration with Biogen, has started a Phase 1/2 clinical study of ISIS-SOD1Rx (BIIB067), one of the drug candidates to come out of the strategic partnership between the two companies, in adult patients diagnosed with amyotrophic lateral sclerosis (ALS). ISIS-SOD1Rx’s mode of action relies on ISIS antisense technology that targets at…