Marisa Wexler, MS,  senior science writer—

The European Commission has approved Qalsody (tofersen) as a treatment for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene, known as SOD1-ALS. Qalsody was specifically granted marketing authorization under exceptional circumstances — a pathway recommended when the benefit-to-risk assessment for a therapy is favorable, but…

By taking advantage of a molecular interaction between the disease-related protein TDP-43 and another, innocuous protein, scientists have found a novel strategy that potentially may be used as a treatment for amyotrophic lateral sclerosis (ALS). “Importantly, this interaction could be key to unlocking a treatment not just for ALS…

Scientists at Broken String Biosciences and the Francis Crick Institute are teaming up to study how genomic instability, or an increased tendency to DNA mutations, contributes to amyotrophic lateral sclerosis (ALS). The findings may shed light on the mechanisms leading to sporadic cases of ALS, the term…

Troriluzole, a new formulation of the approved amyotrophic lateral sclerosis (ALS) treatment riluzole, can be taken with or without food and appears to have better pharmacological properties than approved versions of the medication that allow once daily administration. That’s according to data from three studies in healthy volunteers presented…

Stem cell therapy, specifically treatment with mesenchymal stem cells or MSCs, did not substantially alter disease progression among people with amyotrophic lateral sclerosis (ALS), according to new data from a Phase 2 clinical trial. However, there was a subset of patients who did appear to experience notable benefits from…

In people who experience a traumatic brain injury, or TBI — which is linked to a greater likelihood of developing amyotrophic lateral sclerosis (ALS) — blocking the activity of a protein called KCNJ2 may lower the risk of ALS, according to new research done in lab models. “Targeting KCNJ2…

Taking part in certain recreational activities, including gardening, golfing, woodworking, and hunting, may increase the risk of developing amyotrophic lateral sclerosis (ALS), especially for men, according to a new study. While the causes of ALS still are not well understood, several risk factors have been identified. There’s evidence…

Blocking a protein called PTP sigma led to better nerve survival and motor function in a mouse model of amyotrophic lateral sclerosis (ALS) caused by mutations in the C9ORF72 gene — leading the researchers to highlight this strategy for potentially treating this genetic ALS form. Data from cell models…

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for people with amyotrophic lateral sclerosis (ALS), has failed to meet its primary goal of slowing disease progression in an ongoing Phase 3 clinical trial designed to confirm its effectiveness. That’s according to new data from the trial, dubbed PHOENIX,…

New data show that blood-based measures, including levels of markers of nerve damage and chemical DNA modifications, can help predict which individuals with amyotrophic lateral sclerosis (ALS) will have faster disease progression over time. However, because the disease course may be altered with Radicava or Radicava ORS —…