The accumulation of TDP-43, a widely expressed nuclear protein that binds both DNA and RNA, causes RNA instability in nerve cells derived from patients with amyotrophic lateral sclerosis (ALS). That leads to disruption of energy and protein production, and eventually causes cells’ death. Those findings were described in the…

The active molecule of Petinutin (methsuximide), an anti-epileptic medication, eased motor deficits, extended the lifespan, and showed potent neuroprotective effects in a worm model of amyotrophic lateral sclerosis (ALS), suggesting a potential new treatment strategy for multiple neurodegenerative diseases, a study reports. The study, “α-Methyl-α-phenylsuccinimide ameliorates neurodegeneration…

CannTrust is getting ready to launch a clinical trial to assess the effectiveness of its cannabidiol (CBD) oil capsules on slowing the progression of amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MNDs). This new study is the result of a collaboration between Canada-based CannTrust and…

Treatment of amyotrophic lateral sclerosis (ALS) with investigational compound ibudilast (MN-166) as an add-on to Rilutek (riluzole) improves patients’ functional activity, quality of life, and muscle strength, data from MediciNova’s Phase 2 trial show. MediciNova is now recruiting participants for a Phase 1/2 biomarker trial (…

Xeomin (incobotulinumtoxinA) has been approved by the U.S. Food and Drug Administration for the treatment of chronic sialorrhea, or excessive drooling, a common condition in amyotrophic lateral sclerosis (ALS) patients. Merz Neurosciences, a division of Merz North America, recently announced that its supplemental biologics license application (sBLA) for Xeomin was…

Rare gene mutations in amyotrophic lateral sclerosis (ALS) will be increasingly identified with large-scale, international studies, findings from Project MinE suggest. The study, “Project MinE: study design and pilot analyses of a large-scale whole-genome sequencing study in amyotrophic lateral sclerosis,” was published in the European…

NurOwn, an amyotrophic lateral sclerosis (ALS) treatment candidate, will not be made available under the new Right to Try Act at this time due to a lack of funding alternatives for patients, BrainStorm Cell Therapeutics announced in a press release. The company said it reached this decision after carefully evaluating the Right to…

Flex Pharma announced that it is stopping Phase 2 clinical trials of its investigational compound to ease muscle cramps, FLX-787, in patients with amyotrophic lateral sclerosis (ALS) and Charcot-Marie-Tooth disease (CMT). The company’s decision was based on tolerability concerns in each study with the oral disintegrating tablet formulation at 30…

A new portable system provides insights into the altered tongue, lip, and jaw motion in amyotrohic lateral sclerosis (ALS) patients with a speech disorder called dysarthria. The instrument could be valuable for both diagnosis and treatment of speech impairment. The study, “Articulatory Range of Movement…

Two enzymes called USP5 and USP13 were found to have an important role in fighting back against toxic “stress granules,” a study reports. The study, titled “Deubiquitylases USP5 and USP13 are recruited to and regulate heat-induced stress granules through their deubiquitylating activities,” and published in the …