Patricia Inácio, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NPT520-34, an investigational anti-neuroinflammatory molecule, for the treatment of amyotrophic lateral sclerosis (ALS), Neuropore Therapies announced. “We are very pleased with receiving this orphan drug designation from the FDA’s Office of Orphan Product Development for NPT520-34…

More than 50% of the cases of amyotrophic lateral sclerosis (ALS) disease are linked with a genetic cause, and first-degree relatives of an ALS member — especially daughters of ALS mothers — show the highest risk to inherit it, according to an Irish population-based study. The study “Lifetime Risk…

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 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…

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…

Researchers have created the first entirely human small chip that is able to replicate some of the cell-cell interactions occurring in the peripheral human nervous system, according to a recent study. The Nerve-on-a-Chip platform, developed by AxoSim using organ-on-chip technology, promises to help researchers understand, faster and at…

Researchers identified small-molecule compounds that help to prevent the build-up of stress-induced clumps of TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS). These findings, although preliminary, may lead to new therapies for ALS. The work “Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in…

Nanoparticles may help enhance the therapeutic potential of otherwise difficult-to-deliver agents, such as curcumin, in diseases affecting the central nervous system, including amyotrophic lateral sclerosis (ALS), a review study shows. The review, “Curcumin-loaded nanoparticles: a novel therapeutic strategy in treatment of central nervous system disorders,” was published in the…

A Phase 2b clinical trial assessing the efficacy and safety of H.P. Acthar Gel, an experimental injectable therapy for amyotrophic lateral sclerosis (ALS), has enrolled half of its anticipated participants so far and is still open for recruitment. The trial, called PENNANT (NCT03068754), intends to enroll roughly…