News

Researchers have identified genes that promote amyotrophic lateral sclerosis (ALS), using the gene-editing technology CRISPR-Cas9. The findings represent not only another piece of information to understand the molecular mechanisms triggering ALS, but also support the newly identified genes as potential targets for future therapeutics. The study “CRISPR–Cas9 screens in human cells…

Researchers have made advances in understanding how angiogenin, a key protein associated with amyotrophic lateral sclerosis (ALS), enters and is transported inside nerve cells. Although the mechanisms behind those events were found to be more complex than previously thought, the findings may help to find new targets and new therapies…

Nerve cells that control movement change their size as ALS progresses, becoming larger than normal at first but then ending up much smaller, a mouse study showed. An important finding was that the changes can prevent nerve cells from facilitating communication between the brain and muscles. The research provided scientists with…

Genervon Biopharmaceuticals says its efforts to develop treatments for ALS and other neurodegenerative diseases continue to focus on a factor that regulates stem cells’ evolution into nerve cells. It continues working on Phase 3 clinical trial plans, it added. The company outlined its treatment-development strategy at the…

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

Scientists have gained greater insight into the structure of the nucleolus, a cellular organelle that has been implicated in diseases such as amyotrophic lateral sclerosis (ALS). By honing in on the molecular events surrounding ALS, scientists can start to understand how the disease develops, which could lead to new…

The more a person is exposed to diesel exhaust, the greater the chance they will develop ALS, a Harvard University study on Denmark reports. The team will present the findings at the American Academy of Neurology‘s 70th Annual Meeting in Los Angeles, April 21 to 27. The presentation is titled “…

German researchers have found that attention deficit hyperactivity disorder is not more common in ALS patients than in the general population, despite previous suggestions of a link between them. The study, “Lack of an association between attention-deficit/hyperactivity disorder (ADHD) and amyotrophic lateral sclerosis (ALS),” appeared in the…

A Phase 2/3 clinical trial showed that arimoclomol has the potential to slow ALS patients’ functional decline, according its maker, CytRx Corporation. Arimocolomal did better than a placebo at slowing the decline in patients with a rapidly worsening disease, although the differences were not statistically significant, researchers said. It was also…

Some immune cells in the brain can protect it from the damaging effects of faulty TDP-43 protein, preventing the progression of amyotrophic lateral sclerosis in mice, University of Pennsylvania researchers report. The findings challenged a long-held assumption that the immune cells play a role in damaging nerve cells, the team…