AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash. AVXS-101 is the…
Lack of an enzyme prevents movement nerve cells from developing properly, suggesting that the shortage may play a role in ALS, a Northwestern University study reports. The study in the journal Cell Stem Cell was titled “Dissecting the Functional Consequences of De Novo DNA Methylation Dynamics in Human Motor Neuron Differentiation and…
Biotech investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments have joined efforts by the new private biotech company QurAlis to search for a cure for amyotrophic lateral sclerosis (ALS). Research has shown that ALS is a spectrum of disorders with diverse underlying mechanisms. Similar to…
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…
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…
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