Wave Life’s WVE-004 Shows Proof of Concept in Cell, Mouse Models

WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, has demonstrated proof-of-concept efficacy in cell and animal models, a new study shows. The nucleic acid-based therapy was able to “potently” reduce the toxic RNA molecules and small proteins…

“Take my hand, precious Lord, Lead me home. … When my life is almost gone, Hear my cry, hear my call, Hold my hand lest I fall.” Although I fall light-years shy of the realm of gospel music aficionado, certain songs of that genre move me like no selection from…

Merit E. Cudkowicz, MD,  is the winner of the inaugural Target ALS Rebecca Luker Courage Award in recognition of her work and efforts to help  amyotrophic lateral sclerosis (ALS) patients and the ALS community. Cudkowicz is the chief of neurology at Massachusetts General Hospital, a professor…

Did you know that May has been ALS Awareness Month for the past 30 years? Yes, that’s right, 30 years. Some probably think ALS awareness activities began in 2014 with the ice bucket challenge. At least that’s when many first pronounced the letters A-L-S before dumping a bucket of water…

More than 5,000 people are diagnosed with amyotrophic lateral sclerosis (ALS) each year, and the out-of-pocket costs of care are an estimated $250,000 for each person with the disease, according to the ALS Association. These are just a couple of the statistics about ALS that advocates, organizations, and…

Treatment with Coya Therapeutics‘ ALS001, a regulatory T-cell (Treg) therapy being developed for amyotrophic lateral sclerosis (ALS), was found to reduce markers of oxidative stress — a type of cell damage — and inflammation in a small early trial. The experimental Treg therapy was tested in a Phase 1…

An international team of researchers is asking adults with amyotrophic lateral sclerosis (ALS) and their family caregivers to complete an online survey about their experiences with stigma around the disease. The results are expected to help researchers better understand negative attitudes and untrue beliefs about ALS faced by patients…

Editor’s note: This column discusses suicide. Please find resources for help at the end of the column. In the “Safety Training” episode of the sitcom “The Office,” Michael Scott, the boss, wants to prove that working upstairs in the office is just as risky as working in the…

Researchers at the University of Michigan are focused on establishing cause and effect relationships between environmental and occupational exposures with amyotrophic lateral sclerosis (ALS). They hope this information will shed light on the mechanisms behind the disease and identify modifiable risk factors, which may have implications in preventing ALS.

“I been in the right place, but it must have been the wrong time, I’d have said the right thing, but I must have used the wrong line, I’d have took the right road, but I must have took a wrong turn, Would have made the right move, but I…