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…

Newly identified mutations in the KIF5A gene were recently found to contribute to amyotrophic lateral sclerosis (ALS), the result of a large-scale collaborative study involving more than 250 researchers from several of the world’s renowned ALS research labs. John Landers, PhD, professor at the University of Massachusetts Medical…

The U.S. prevalence of amyotrophic lateral sclerosis (ALS) in 2014 was 5.0 per 100,000 people, which was unchanged from 2013, according to researchers. Men, whites, and people 60 and older continue to show the highest rates. The study, “Prevalence of Amyotrophic Lateral Sclerosis — United States, 2014,” was published in…

Prothena and Celgene are teaming up to develop treatments for a range of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). The collaboration will focus on therapies that can target proteins such as tau and TDP-43 that scientists have linked to these disorders. “The programs we have chosen to collaborate…

Patients with amyotrophic lateral sclerosis (ALS) show higher rates of sleep apnea that is often accompanied by nocturnal hypoventilation, according to a retrospective study. Researchers also found that a method called transcutaneous capnography is able to reliably detect nocturnal hypoventilation, or insufficient breathing, in ALS patients. The…