Occupational exposure to lead — a heavy metal — prior to disease onset is associated with a more severe disease progression and lower survival among people with amyotrophic lateral sclerosis (ALS), according to a recent study. “In survival analysis of an international cohort [group] of ALS cases, our results…

The experimental therapy WVE-004 appears to be engaging its intended target and reducing the amount of toxic proteins in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene, according to early data from the FOCUS-C9 clinical trial. The trial is currently recruiting adults,…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

“‘The problem is all inside your head,’ she said to me, ‘The answer is easy if you take it logically,’ … She said, ‘It grieves me so to see you in such pain, I wish there was something I could do to make you smile again.’ I said, ‘I appreciate…

ALPHA-0602, an investigational gene therapy for amyotrophic lateral sclerosis (ALS), can successfully increase progranulin levels and reduce the toxicity of TDP-43 protein aggregates in cell cultures and mouse models, Alpha Cognition, the therapy’s developer, announced. “These new insights from our preclinical research further support the development of ALPHA-0602…

Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…

I got away for a couple of nights during spring break with my 12-year-old son, Isaac, and 16-year-old daughter, Sara. It was good to have a mini-vacation, although I was sad my husband, Todd, couldn’t join us because his ALS progression makes travel too difficult. Three years ago, we all…

Minnesota lawmakers have passed legislation that will provide $25 million for amyotrophic lateral sclerosis (ALS) research and to support ALS caregivers in the state. Under the law (SF 3372), the Minnesota Office of Higher Education will receive $20 million to award research grants to scientists studying all areas of…

A CRISPR-based gene editing system could be used to reduce the activity of genes associated with amyotrophic lateral sclerosis (ALS) and Huntington’s disease, a new study shows. The study, “Targeted gene silencing in the nervous system with CRISPR-Cas13,” was published in Science Advances. CRISPR is a strategy that…

“I know all too well it don’t come easy, The chains of the world they seem to movin’ tight. I try to walk around, if I’m stumbling so come, Tryin’ to get up but the doubt is so strong, There’s gotta be a winning in my bones. I am looking…