When the TDP-43 protein is less able to form two-protein complexes called dimers, it appears to be more likely to form the toxic clumps seen in amyotrophic lateral sclerosis (ALS), a study reports. As these clumps are found in about 97% of all ALS cases, a better understanding of the…
Scientists have devised a technique for transplanting healthy motor neurons — the nerve cells that are lost in amyotrophic lateral sclerosis (ALS) — in a mouse model with “highly aggressive” disease. Transplanted motor neurons, given stimulation, were able to form healthy connections with muscle cells to control the animals’ muscle…
Akava Therapeutics will soon launch a Phase 1 clinical trial to test AKV9, its investigational therapy for amyotrophic lateral sclerosis (ALS), in healthy people. The trial follows the clearance of Akava’s investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). It will be AKV9’s…
My late husband, Jeff, made a dream come true for me. When I was a kid, my dad would take our family on our small powerboat to explore the waters near my hometown of Annapolis, Maryland. If we had enough time and the weather cooperated, we’d take a…
One evening last week after I’d finished cooking dinner, I grew concerned. My almost 14-year-old son had gone out to ride his dirt bike on an off-road vehicle (ORV) trail that ran next to our property. He’d planned to go only to a nature area a couple miles away and…
BL-001, an investigational gut-targeted therapy being developed for people with amyotrophic lateral sclerosis (ALS) and Dravet syndrome, was found to be safe and well tolerated among healthy volunteers in a Phase 1 clinical trial. Findings overall indicate that Bloom Science‘s BL-001 can be given safely to humans at…
AstroRx is an investigational cell-based therapy that is being tested in early clinical trials for amyotrophic lateral sclerosis (ALS). It aims to slow ALS progression by preventing further damage to nerve cells.
Elevated blood levels of the Cystatin C protein are significantly associated with faster disease progression and shorter survival in people with amyotrophic lateral sclerosis (ALS), according to a study in China. The protein may serve as a biomarker to predict disease outcomes in people with ALS and identify those…
Last week, I sat in the dentist’s chair filled with gratitude. I know. It’s bonkers. But I couldn’t help myself. I’m grateful knowing that if I have to live with ALS, it’s happening at a time when advances in technology and accommodations help me deal with its many challenges.
Early treatment with Radicava (edaravone), an approved therapy for amyotrophic lateral sclerosis (ALS), significantly reduced the risk of hospitalization, need for ventilatory support, or death compared with delaying treatment for six months. That’s according to analyses of data from Study 19 (NCT01492686), the Phase 3 clinical trial…
