Steve Bryson, PhD, science writer —

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…

The loss of chemical modifications in the regulatory sequence of the TARDBP gene, called DNA methylation, seems to contribute to increased levels and clumping of the TDP-43 protein in the motor cortex of people with amyotrophic lateral sclerosis (ALS), a study found. As methylation in this gene — which…

A small open-label study evaluating the epilepsy medication perampanel in adults with amyotrophic lateral sclerosis (ALS) was halted due to adverse events that affected behavior. Despite the findings, larger clinical trials investigating perampanel in ALS patients are ongoing, which might determine if the medication contributed to behavioral side effects. The…

CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), failed to prevent motor neurons loss and lung function decline relative to a placebo, according to top-line data from the RESCUE-ALS Phase 2 clinical trial. The trial, in other words, did not meet its primary…

Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…

A detailed atlas of the various types of cells that populate the motor cortex, the brain region that controls voluntary movement and is damaged in people with amyotrophic lateral sclerosis (ALS), was created by a worldwide consortium of researchers. The long-term goal of the group, which involves hundreds of…

An investigational treatment for amyotrophic lateral sclerosis (ALS), SBT-272 was found to sustainably reach different brain regions, and to protect mitochondria — a cell’s energy source — from TDP-43 toxic aggregates in a mouse model of the disease. “We are excited about the promise of SBT-272 as a potential therapeutic…

Diminished activity in the C9orf72 gene that is linked with amyotrophic lateral sclerosis (ALS) affected the neuromuscular junction — where nerve and muscle cells connect — in a zebrafish model, establishing the model as a disease research tool and the gene’s role in ALS symptoms. Zebrafish in this C9orf72 loss-of-function model…

Men who played professional soccer were nearly four times more likely to develop a neurodegenerative disorder, including a motor neuron disease such as amyotrophic lateral sclerosis (ALS), than men of similar age in the general population, a long-term analysis found. Risk was greatest in players with longer careers and…

Using motor neurons derived from people with amyotrophic lateral sclerosis (ALS) is a promising approach to screen for molecules that prevent certain disease mechanisms, and to identify new targets for therapies, according to a recent study. Two classes of therapies were identified that might lessen the excessive firing of motor…