Marisa Wexler, MS,  senior science writer—

Changes in how the SOD1 protein associates with metal molecules seem to be key for how mutations affecting this protein drive amyotrophic lateral sclerosis (ALS), according to a study using a novel imaging technique. Findings represent “a very early step towards” new ALS treatments, while also showing “an exciting…

One year of treatment with the experimental oral therapy PrimeC led to improved iron accumulation in people with amyotrophic lateral sclerosis (ALS), according to new clinical trial data announced by PrimeC’s developer, Neurosense Therapeutics. Previous results showed that people who received PrimeC for one year experienced significantly slower…

Clene will soon meet with the U.S. Food and Drug Administration (FDA) to discuss the potential pathway to accelerated approval for CNM-Au8, the company’s experimental therapy for amyotrophic lateral sclerosis (ALS). Ahead of the meeting, the company has submitted new data to the agency, including the…

A mutation in the IGFBP7 gene is associated with a very rare phenomenon in which people with amyotrophic lateral sclerosis (ALS) have a reversal in disease progression and experience partial or full recovery, a new study reports. Because the mutation results in less production of the resulting protein IGFBP7,…

Treatment with a high dose of ILB, an experimental injection therapy being developed by Tikomed, was well tolerated and appeared to slow the progression of amyotrophic lateral sclerosis (ALS) in a small clinical trial in the U.K. That’s according to data from the exploratory Phase 2 study…

In amyotrophic lateral sclerosis (ALS), nerve cells in the spinal cord responsible for inhibiting motor neurons are lost in the early stages of the disease, before the motor neurons themselves, according to a new study done in a genetic mouse model of ALS. The researchers also found that excitatory…

After up to 3.5 years on the experimental oral treatment CNM-Au8, the chances of survival for people with amyotrophic lateral sclerosis (ALS) in the open-label extension of the HEALEY ALS platform trial was nearly 60% higher than for a group of patients given a placebo in previous studies.

The U.S. Department of Defense (DoD) has awarded more than $2 million to help Pathmaker Neurosystems conduct a second clinical trial of its MyoRegulator — an experimental, noninvasive nerve modulating device — in people with amyotrophic lateral sclerosis (ALS). “We are very pleased to have…

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…