Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…
A molecule that blocks the regulatory RNA protein HuR, called SRI-42127, strongly reduces brain inflammation driven by activated glial cells, key players in neurodegenerative diseases like amyotrophic lateral sclerosis, a preclinical study reported. This inhibitor may be of promise in treating ALS and other diseases driven by inflammation in the brain. “Our…
Numotion has partnered with Team Gleason — the nonprofit started by former pro football player Steve Gleason after his 2011 amyotrophic lateral sclerosis (ALS) diagnosis — to improve access to advanced equipment and technology for ALS patients and their families. The partners also will focus on developing mobility and…
Age, socioeconomic status, and co-existing conditions are among the factors that can lead to a need for assisted ventilation in people with amyotrophic lateral sclerosis (ALS), research from Korea reports. The study, “Factors associated with assisted ventilation use in amyotrophic lateral sclerosis: a nationwide population-based study in Korea,”…
Certain blood biomarkers of lipid, or fat, metabolism — typically measured to determine a person’s risk of cardiovascular disease — appear to protect against developing amyotrophic lateral sclerosis (ALS), a study of more than 500,000 adults reported. Specifically, researchers found that people with higher levels of high density lipoprotein (HDL)…
Total job- and leisure-related physical activity over a lifetime is not a risk factor for amyotrophic lateral sclerosis (ALS), but heavy physical activity on the job is associated with a nearly two-times greater risk of ALS, a study in Germany shows. Also, ALS patients reporting pre-diagnosis and current moderate…
Using a new laboratory model of brain cells, researchers demonstrated that cells with mutations associated with amyotrophic lateral sclerosis (ALS) show early signs of DNA damage and other forms of cellular stress. Results suggest that blocking these cellular stress processes may hold promise in treating ALS. The study, “…
A Phase 2 study of the investigational antibody AT-1501 in treating amyotrophic lateral sclerosis (ALS) has completed patient enrollment in three of its four dosing groups, and is likely to be fully enrolled by year’s end, its developer, Eledon Pharmaceuticals, announced. A total of 54 ALS patients diagnosed in the…
New early clinical study results support the potential of GN-K01, an investigational antibody being developed by GeNeuro, as a treatment for amyotrophic lateral sclerosis (ALS). The therapy may enter clinical trials as early as 2022, according to the company. The antibody targets a viral protein, called HERV-K ENV,…
Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…
