The first patients to complete a Phase 2 clinical trial have chosen to continue treatment in an open-label extension (OLE) study of Amylyx Pharmaceuticals‘ investigative therapy AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid…
Amylyx Enrolls First ALS Patients in Phase 2 Extension Trial of AMX0035
Higher levels of a specialized type of immune cell may help halt the progression of amyotrophic lateral sclerosis (ALS), a study in humans and mice found. These findings were reported in the journal JAMA Neurology, in the study, “Association of Regulatory T-Cell Expansion With Progression of Amyotrophic…
This time around, I really, really tried not to let it happen. But it did. I washed my hands. I avoided all contact with suspicious “others,” and even hauled around a good-sized tub of disinfectant wipes. All my valiant efforts made the world around me a little cleaner,…
EH301, Elysium Health‘s investigational therapy for amyotrophic lateral sclerosis (ALS), was recently granted orphan drug status by the U.S. Food and Drug Administration. Elysium’s application for orphan drug designation included data from a 2017 double-blind, placebo-controlled European pilot study in humans. To expand on the results of the pilot study,…
The Israeli biotechnology company Kadimastem is planning a Phase 1/2a clinical trial of a cell therapy for ALS after regulators gave it the go-ahead. Kadimastem’s therapy, AstroRx, consists of cells called astrocytes that are injected into cerebrospinal fluid. The star-shaped cells, which surround nerve cells in the brain and spinal cord, are derived…
BrainStorm Cell Therapeutics is adding Canadians to a Phase 3 U.S. clinical trial of its ALS stem cell therapy NurOwn. It decided to add the Canadians when a number of them expressed interest in participating, BrainStorm said. The trial (NCT03280056) is investigating the safety and effectiveness of repeated doses…
The brain’s smallest blood vessels stimulate the growth of spinal cord nerve cells at an early stage of their development, researchers at Southern California’s Cedars-Sinai group report. Their discovery, made with a tiny biological chip, could help shed light on the causes of amyotrophic lateral sclerosis (ALS). The article they wrote…
I don’t know about your email inbox, but mine often overflows with news alerts. I can usually buzz through them fairly quickly, but last week, three items caught my eye and had me clicking through to learn more. Exercise is safe for ALS patients Until my ALS diagnosis…
A novel mouse model and a new method of analyzing these mice led researchers to the discovery of key molecular changes in the early stages of amyotrophic lateral sclerosis (ALS), opening up the possibility of new therapy targets. These new innovations could be valuable tools for researchers to continue to…
Newly identified mutations in the KIF5A gene were recently found to contribute to amyotrophic lateral sclerosis (ALS), the result of a large-scale collaborative study involving more than 250 researchers from several of the world’s renowned ALS research labs. John Landers, PhD, professor at the University of Massachusetts Medical…
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