News

A branch of the European Medicines Agency (EMA) has recommended that Amylyx‘s investigational oral compound AMX0035 be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Orphan designation is given to medicines in Europe with the potential to be safe…

Reducing the excess firing of motor neurons in an animal model of amyotrophic lateral sclerosis (ALS) prevents the death of nerve cells and significantly delays the onset of motor symptoms, a recent study has found. Because the scientific approach made use of two techniques that have been studied extensively…

A team at the Rowan University School of Osteopathic Medicine has received a $1.6 million grant to study the role of the SOD1 protein — whose gene is often mutated in amyotrophic lateral sclerosis (ALS) — in the communication between the gut and the brain. The National Institutes…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

The Muscular Dystrophy Association (MDA) is hosting a Facebook Live event on May 1 concerning precautions and best practices needed to protect the amyotrophic lateral sclerosis (ALS) community during the COVID-19 outbreak. The half-hour event, which kicks off ALS Awareness Month, is to begin at…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

A weak link only between a person’s current smoking study and greater risk of amyotrophic lateral sclerosis (ALS) was seen by scientists in a U.K. study, with no evidence that lifetime smoking exposure affects such risk. The researchers believe that this weak association is likely a “false-positive,” and there…

Injecting tiny particles carrying adapalene — an activator of the retinoic acid signaling pathway — to the brain and spinal cord of mice with amyotrophic lateral sclerosis (ALS) can prolong animals’ survival, improve their motor function, and prevent neurodegeneration, a study shows. The study, “…

A  way of examining multiple genes or the entire human genome at the same time, called next-generation sequencing (NGS), is a promising tool for diagnosing amyotrophic lateral sclerosis (ALS) in routine clinical practice, a review study suggests. This method’s approach is faster and less expensive than the standard…