News

Amyotrophic lateral sclerosis (ALS) patients with mild to moderate disease progression show an ability to drive that’s comparable to other adults, despite weaknesses with movement and cognition, a small study using a driving simulator suggests. The study included only patients who were still driving, and its researchers caution that…

Eight patients will receive jacifusen, an experimental therapy for amyotrophic lateral sclerosis (ALS) caused by FUS gene mutations, under a joint effort from the ALS Association, Project ALS, and Columbia University’s Eleanor and Lou Gehrig ALS Center. The new clinical research program, which will be supported…

Studies in animal models investigating mitochondrial abnormalities in amyotrophic lateral sclerosis (ALS) found that therapies targeting mitochondria — the powerhouses of cells — are consistently effective in prolonging survival, a review concluded. The review study, “Targeting…

Like many couples, Joe and Linda Lacroix of Milton, Vermont, spent much of their lives working, raising families, maintaining their home, and planning for the future. Unlike most, however, there was always a dark cloud looming on their horizon — a gene for Huntington’s disease carried by Joe’s mother.

Skin cells from people with amyotrophic lateral sclerosis (ALS) have altered metabolisms and increased levels of a type of cellular stress called oxidative stress, a new study shows. This may indicate new treatment targets for ALS. The study, “ALS skin fibroblasts reveal oxidative stress and ERK1/2-mediated…

A machine learning algorithm has shown that depression and a perceived lower quality of life are significant predictors of high caregiver burden among those who care for patients with amyotrophic lateral sclerosis (ALS), a study has found. The study, “Prediction of caregiver burden in amyotrophic lateral sclerosis:…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

There are differences in the way the fluid around the brain and spinal cord moves in people with amyotrophic lateral sclerosis (ALS), and understanding these could have implications for the development of future therapies, a study suggests. The study, “Non-invasive MRI quantification of cerebrospinal fluid dynamics…

A gene editing technology based on CRISPR was able to slow the progression of amyotrophic lateral sclerosis in a mouse model, a new study showed, demonstrating the approach’s potential for gene therapy in people with ALS. The study, “Treatment of a Mouse Model of ALS by…

The European Medicines Agency (EMA) granted orphan drug status to Cytokinetics‘ reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The move follows fast track designation granted in December by the U.S. Food and Drug Administration for the same indication, and provides the…