News

People with amyotrophic lateral sclerosis (ALS) have significantly lower levels of a small molecule called microRNA-335-5p (miR-335-5p), which promotes the degradation of mitochondria and nerve cell death, a study shows. These findings suggest that dysregulation of miR-335-5p may contribute to neurodegeneration in ALS patients, which may be useful…

Fasudil, approved in Japan for treating stroke patients, also is well-tolerated and prevents disease worsening in people with amyotrophic lateral sclerosis (ALS), according to a report of three patients who received the medicine under compassionate use. However, the effects were transient, and all three patients experienced either a decline…

Mutations in the TBK1 gene — often associated with amyotrophic lateral sclerosis (ALS) — seem to have a dual role in progression of the disease, causing it to emerge earlier in animals models of ALS, but then slowing in later stages. This paradoxical role, according to a results…

By blocking the activity of the proteasome — a complex of enzymes responsible for the destruction of unnecessary or damaged proteins — dipeptide repeat (DPR) proteins associated with C9ORF72 gene repeat expansions (the most common genetic cause of amyotrophic lateral sclerosis) can travel between nerve cells…

The onset and early progression of motor and cognitive symptoms, specific by disease type to each amyotrophic lateral sclerosis (ALS) patient, are determined by age and sex, and to some extent by mutations in ALS genes, a large data study has found. The study, “ALS phenotype is influenced…

AB Science plans to launch a Phase 3 clinical trial testing the safety and effectiveness of its tyrosine kinase inhibitor masitinib as an add-on treatment for people with amyotrophic lateral sclerosis (ALS). Its announcement follows U.S. Food and Drug Administration (FDA) approval of the company’s investigational new…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. The 30-minute session can be accessed on the MDA’s Facebook page, and starts at 6 p.m. EST. People with…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

A single brain area previously thought to control only the movement of the hands and arms also may control movement across all four limbs, a study found. This discovery opens new avenues for the use of brain-computer interfaces in people who have lost control of their arms and legs,…