News

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…

Variations of the TP73 gene could increase a person’s susceptibility to developing the non-inherited version of ALS, a study suggests. University of Utah researchers will present the study at the American Academy of Neurology annual meeting in Los Angeles at 4:30 p.m. Pacific time on April 24. The title is “…

NurOwn, a cell therapy being developed by Brainstorm Cell Therapeutics, is able to maintain its therapeutic effects by significantly regulating the levels of a type of small RNA molecule linked to amyotrophic lateral sclerosis (ALS), a study suggests. According to data from a Phase 2 trial, the investigative therapy can…

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…

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…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of 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…