Marisa Wexler, MS,  senior science writer—

A CRISPR-based gene editing system could be used to reduce the activity of genes associated with amyotrophic lateral sclerosis (ALS) and Huntington’s disease, a new study shows. The study, “Targeted gene silencing in the nervous system with CRISPR-Cas13,” was published in Science Advances. CRISPR is a strategy that…

A novel system that involves electrodes implanted in the brain allowed a 34-year-old man with amyotrophic lateral sclerosis (ALS) who had completely lost the ability to move voluntarily — known as a “locked-in” state — to communicate with his family again. Using the system, the man was able to…

Two independent teams of scientists have unraveled the molecular mechanism by which abnormal localization of the TDP-43 protein, which is characteristic of amyotrophic lateral sclerosis (ALS), leads to reduced levels of another protein, called UNC13A, that is important for nerve cell function. The results also show that certain variations…

Treatment with the cell therapy NurOwn may slow the progression of amyotrophic lateral sclerosis (ALS) for those with a specific variation in the UNC13A gene that is linked with a high risk of the disease. Merit Cudkowicz, MD, chief of neurology at the Massachusetts General Hospital, shared the…

Treatment with Clene Nanomedicine’s experimental medicine CNM-Au8 may decrease the risk of disease progression or death in people with early amyotrophic lateral sclerosis (ALS), according to new analyses from the RESCUE-ALS clinical trial. Data also suggest that the therapy helped to prevent the loss of motor neurons in…

Exposure to certain toxic compounds — including beta-N-methylamino-L-alanine (BMAA), formaldehyde, and heavy metals like manganese, mercury, and zinc — increases the likelihood of developing amyotrophic lateral sclerosis (ALS), according to a review paper. Its authors argue that with BMAA — a toxic compound made by algae — enough evidence…

Mutations in the FUS gene, which cause some forms of amyotrophic lateral sclerosis (ALS), result in a circular RNA molecule called circ-Hdgfrp3 being pulled into toxic protein clumps that form in nerve cells, according to a study. This is the first time that a circular RNA has been shown…

The Cullen Education and Research Fund (CERF), a private philanthropy based in London, has awarded more than $800,000 to support two U.S. research projects that are working to improve life for people with amyotrophic lateral sclerosis (ALS). The CERF Medical Engineering Prize, worth €500,000 (about $570,000), was awarded to…

A team of scientists in the U.K. and Japan has determined the structure of aggregated TDP-43, the protein whose abnormal clumps are characteristic of amyotrophic lateral sclerosis (ALS). Their work, reportedly the first to reveal the molecular structure of aggregated TDP-43, identified a “double-spiral fold” of the protein in patients’ brain…

A decision is expected by May from the U.S. Food and Drug Administration (FDA) on an application seeking approval of an oral formulation of edaravone (MT-1186) — one with a similar clinical profile to Radicava — as a treatment for amyotrophic lateral sclerosis (ALS). The FDA is now…