Genetic mutations in two previously unrecognized genes, the microtubule-associated protein tau (MAPT) and BNIP1 genes, are associated with elevated risk for amyotrophic lateral sclerosis (ALS). The study with that finding “Selective genetic overlap between amyotrophic lateral sclerosis and diseases of the frontotemporal dementia spectrum,” was published in the…

NMD Pharma has obtained $47 million in financing from new investors to continue developing treatments for ALS and other neuromuscular disorders. The Danish company is focusing on small-molecule inhibitors of a muscle-related chloride ion channel. It has created a screening platform to identify potential inhibitors of the ClC-1 ion channel.

Health Canada accepted Mitsubishi Tanabe Pharma’s new drug submission (NDS) for edaravone as an intravenous treatment option for amyotrophic lateral sclerosis (ALS). Edaravone is currently approved as a treatment for ALS in Japan, South Korea, and the United States (where it is marketed by Mitsubishi Tanabe as Radicava).

Rilutek (riluzole) extends survival of patients with amyotrophic lateral sclerosis (ALS) who are at  advanced stages of the disease, rather than preventing disease progression is early stages. Those findings were reported by a team led by King’s College London researchers and published in The Lancet Neurology. The…

AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash. AVXS-101 is the…

Biotech investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments have joined efforts by the new private biotech company QurAlis to search for a cure for amyotrophic lateral sclerosis (ALS). Research has shown that ALS is a spectrum of disorders with diverse underlying mechanisms. Similar to…

The first patients to complete a Phase 2 clinical trial have chosen to continue treatment in an open-label extension (OLE) study of Amylyx Pharmaceuticals‘ investigative therapy AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid…

Higher levels of a specialized type of immune cell may help halt the progression of amyotrophic lateral sclerosis (ALS), a study in humans and mice found. These findings were reported in the journal JAMA Neurology, in the study, “Association of Regulatory T-Cell Expansion With Progression of Amyotrophic…

This time around, I really, really tried not to let it happen. But it did. I washed my hands. I avoided all contact with suspicious “others,” and even hauled around a good-sized tub of disinfectant wipes. All my valiant efforts made the world around me a little cleaner,…

EH301, Elysium Health‘s investigational therapy for amyotrophic lateral sclerosis (ALS), was recently granted orphan drug status by the U.S. Food and Drug Administration. Elysium’s application for orphan drug designation included data from a 2017 double-blind, placebo-controlled European pilot study in humans. To expand on the results of the pilot study,…