New antisense oligonucleotide (ASO) molecular therapies targeting the most common gene mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia reduced brain disease hallmarks in a mouse study, researchers found. The study, “Stereopure Antisense Oligonucleotides Preferentially Knockdown G4C2 Repeat-Containing C9ORF72 Transcripts: A Potential Therapeutic Approach for the Treatment…

Researchers suggest that patients in the early stage of amyotrophic lateral sclerosis (ALS) may benefit from a personalized program of home-based exercises without a physical therapist’s supervision. The small Japanese study, “Effectiveness of home-based exercises without supervision by physical therapists for patients with early-stage amyotrophic lateral sclerosis: A…

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…

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,…

The brain’s smallest blood vessels stimulate the growth of spinal cord nerve cells at an early stage of their development, researchers at Southern California’s Cedars-Sinai group report. Their discovery, made with a tiny biological chip, could help shed light on the causes of amyotrophic lateral sclerosis (ALS). The article they wrote…

A novel mouse model and a new method of analyzing these mice led researchers to the discovery of key molecular changes in the early stages of amyotrophic lateral sclerosis (ALS), opening up the possibility of new therapy targets. These new innovations could be valuable tools for researchers to continue to…