New research has identified the RPS25 gene as a key player that allows the production of mutated C9orf72 protein — one that is prone to aggregation, or clumps. The gene that codes for this protein is one of the most commonly mutated genes underlying amyotrophic lateral sclerosis (ALS), the…

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…

Cognitive problems have limited impact on overall health-related quality of life (QoL) in people with amyotrophic lateral sclerosis (ALS) — but verbal fluency and space orientation are among the factors linked to patients’ emotional well-being, according to new research. The study, “Cognitive deficits have only…

While rehabilitation programs that include exercise are helpful to amyotrophic lateral sclerosis (ALS) patients, no extra benefit was seen by raising the number of weekly sessions from a standard two to five, a small study from Italy reports. The study, “High‐frequency motor rehabilitation in amyotrophic lateral sclerosis: a randomized…

The gut microbiome — the natural collection of microorganisms living in our guts —  might influence the progression of amyotrophic lateral sclerosis (ALS), recent research suggests. Specifically, researchers found that a metabolite produced by the Akkermansia muciniphila bacteria, called nicotinamide, slowed disease progression and prolonged survival in an ALS mouse…

Concerns over an active pharmaceutical ingredient used in a 2017 study of Nurtec (riluzole) as a bioequivalent medicine were voiced by the U.S. Food and Drug Administration (FDA) in its response to a request for the treatment’s approval for  amyotrophic lateral sclerosis (ALS), its developer, Biohaven Pharmaceuticals,…

The Chinese caterpillar fungus (Hirsutella sinensis), used in traditional Chinese medicine, minimized motor neuron loss and extended the lifespan in a mouse model of amyotrophic lateral sclerosis (ALS), a study has found. The study, “Therapeutic effects of hirsutella sinensis on the disease onset and progression of amyotrophic lateral…

Patient enrollment is now complete, ahead of schedule, for the Phase 3 ORARIALS-01 clinical trial assessing the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). “The completion of enrollment for Orphazyme’s pivotal ALS trial is another great milestone for our clinical development program. We are confident that…

Mallinckrodt Pharmaceuticals has shut down its Phase 2b PENNANT clinical trial assessing the effectiveness and safety of H.P. Acthar Gel, an experimental injectable therapy for amyotrophic lateral sclerosis (ALS). The reason for this decision was an increased incidence of pneumonia among ALS patients receiving H.P. Acthar Gel compared to…

Specific populations of neurons and associated cells in the spinal cord of people with sporadic amyotrophic lateral sclerosis (ALS) may serve as early biomarkers for the disease, a study has found. The newly discovered populations offer scientists a chance to learn more about the first steps underlying ALS and…