Magdalena Kegel,  —

Aggregates of dipeptide repeat proteins produced by the mutant gene C9orf72 — the most common genetic cause of amyotrophic lateral sclerosis — are toxic to neurons through interactions with a protein intended to clear cells of defective proteins, scientists reported. The findings provide a very specific focus for future drug…

Mutations in the C9orf72 gene are the most common genetic cause leading to amyotrophic lateral sclerosis (ALS). Despite this, researchers have not been able to determine what the normal function of the gene really is. Now, scientists from Cedars-Sinai Medical Center in Los Angeles have revealed that the gene contributes to…

Scientists from Rutgers and Stanford universities have developed a potentially revolutionary technique to grow new neurons from adult stem cells and better ensure their survival during transplantation to the brain. The method, involving the use of 3-D scaffolds, may one day help to treat amyotrophic lateral sclerosis (ALS). Published in the…

When the New York State Department of Health‘s Medical Marijuana Program was launched on Jan. 7, 2016, Upper East Side physician Dr. Thomas O’Brien was the first doctor to secure a certification for a patient to be prescribed medical marijuana. He has since helped 200 patients to achieve…

Researchers at The Scripps Research Institute discovered that cells instruct proteins to form aggregates to protect them from toxicity associated with neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). The research team, led by Claudio Joazeiro, associate professor of cell and molecular biology at Scripps, identified an enzyme that…

German researchers have identified a new gene — called NEK1 — that appears to be responsible for familial amyotrophic lateral sclerosis (ALS). Loss of function mutations in the gene were more common in ALS patients than in healthy controls, according to findings published in the journal Brain. Familial ALS…

Portage Biotech and Biohaven Pharmaceuticals recently announced they have received positive feedback from the U.S. Food and Drug Administration (FDA) on their Pre-Investigational New Drug Application (PIND) interaction regarding Biohaven’s investigative drug BHV-0223, intended for treatment of amyotrophic lateral sclerosis (ALS). BHV-0223 is the glutamate modulating compound riluzole, contained in the tablet…

Cytokinetics announced that it has joined with efforts by the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to raise awareness for Rare Disease Day. Cytokinetics is focused on discovering, developing, and commercializing new types of muscle activators, and has developed the amyotrophic lateral sclerosis (ALS) drug candidate…

U.S. veterans who fought in the Persian Gulf War of 1990-91 appear to be at an increased risk for amyotrophic lateral sclerosis (ALS), according to report from the Institute of Medicine (IOM) of the National Academies of Sciences, Engineering, and Medicine. The U.S. Department of Veterans Affairs had requested the review, evaluating and summarizing…

Belgian researchers found the downstream effects of a mutation in the gene C9orf72, identified in 2011 as one of the main genetic factors behind amyotrophic lateral sclerosis (ALS). This specific mutation interferes with nuclear transport of proteins in and out of the cell nucleus and contributes to ALS development. About 10 percent of…