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…
I’d like to send a hearty congratulations to us all! Why the celebration? Well, we’ve survived the first three months of the year in a world that continues to surprise and challenge us. Plus, for many who live with amyotrophic lateral sclerosis (ALS), making it through another three months justifies…
Treatment with Radicava (edaravone) for at least one year significantly reduces the risk of death in people with amyotrophic lateral sclerosis (ALS), prolonging their survival for about six months compared with those not given the therapy, an analysis of U.S. real-life data shows. “Real-world data may provide additional insight…
Synchron’s Stentrode, an innovative, implantable brain computer interface (BCI), was found safe in four amyotrophic lateral sclerosis (ALS) patients and let them communicate and perform daily online tasks by using “just their thought,” according to one-year data from a clinical trial. The results were presented as part of…
There is no substantial evidence supporting the efficacy of Amylyx Pharmaceuticals’ AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), according to a 6–4 vote by a U.S. Food and Drug Administration (FDA) advisory committee. The close vote against the therapy came at the end of a virtual…
An old friend stopped by with a gift of homegrown microgreens. She’s a retired nurse, and she used to help with nighttime care for my husband, Todd, who has ALS. She visited for a while, and we updated her on Todd’s health and decline in lung strength. I…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to InFlectis BioScience’s experimental therapy IFB-088 for amyotrophic lateral sclerosis (ALS). Orphan drug status is given to treatment candidates with the potential to be safe and effective in rare diseases. In the U.S. rare diseases are defined as…
“By the rivers of Babylon Where we sat down And there we wept When we remembered Zion But the wicked carried us away in captivity Required from us a song How can we sing King Alpha song In…
Regulus Therapeutics, in partnership with researchers at Brigham and Women’s Hospital, has initiated studies to evaluate a library of molecules designed to suppress microRNA-155 (miR-155) — a small molecule that regulates the activity of other genes — as a potential treatment for amyotrophic lateral sclerosis (ALS). The agreement, effective…
