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…
Progranulin Gene Therapy Shows Promise in Preclinical Studies
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…
Team Drea Foundation is launching a customizable wellness challenge that seeks to raise $50,000 in 50 days for amyotrophic lateral sclerosis (ALS) research. The 50for50 challenge invites people to set a personal goal related to the number 50, such as 50 pushups a day or 50 acts…
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…
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…
Recent Posts
- Tossing and turning over fears this ALS journey has come to its end
- Experimental ALS therapy QRL-201 shows potential to slow disease decline
- Worried about an inability to multitask? Here’s what helped me.
- MDA launches Shamrocks fundraiser to advance neuromuscular research
- Early-career ALS scientist wins first MDA Research Momentum Award