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…

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…

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…

Scientists who dissected the structure, and mechanism of action, of the protein Cdc48 — dubbed the “Swiss Army knife of cells” — say it can be explored as a therapeutic target for several disorders, including amyotrophic lateral sclerosis (ALS). The findings of the study, “Structure of the…

The Phase 3 REFALS trial exploring the impact of oral levosimendan (ODM-109) in patients with amyotrophic lateral sclerosis (ALS) has completed its target enrollment, the therapy’s manufacturer, Orion, announced. A total of 496 participants were recruited across 104 sites in the United States, Canada, Europe, and Australia. They…

A study in identical twins or triplets found that changes in DNA marks, collectively known as DNA methylation and typically associated with aging, can like genetics play an important role to determining the development of amyotrophic lateral sclerosis (ALS). The four sets of ALS-affected and unaffected siblings carried age-related differences…