A new Phase 3 trial evaluating levosimendan (also known as ODM-109), an oral treatment by Orion for breathing problems in amyotrophic lateral sclerosis (ALS), has recruited its first patients, the Finnish pharmaceutical company announced. The trial, called REFALS (NCT03505021), aims to investigate the potential benefits of prolonged treatment with levosimendan…
News
2 miRNAs Controlling Genes Involved in ALS, and Possible Way of Regulating Them, Identified in Study
Sporadic and familial forms of amyotrophic lateral sclerosis (ALS) are associated with two microRNA molecules that are downregulated — meaning these molecules, involved in the protein production of certain genes, cause less protein to be created —, researchers have discovered. And they found a possible way of “upregulating” these molecules, making…
Xeomin (incobotulinumtoxinA) has been approved by the U.S. Food and Drug Administration for the treatment of chronic sialorrhea, or excessive drooling, a common condition in amyotrophic lateral sclerosis (ALS) patients. Merz Neurosciences, a division of Merz North America, recently announced that its supplemental biologics license application (sBLA) for Xeomin was…
BrainStorm Cell Therapeutics has partnered with the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute for the manufacturing of NurOwn, a therapy candidate for amyotrophic lateral sclerosis (ALS), for BrainStorm’s ongoing Phase 3 trial. This is the second U.S. production site…
Skyhawk Therapeutics and Celgene have agreed to collaborate for five years to discover, develop, and commercialize innovative small molecule treatment options for neurological diseases with high unmet medical needs, including amyotrophic lateral sclerosis (ALS) and Huntington’s disease. Several genetic disorders are caused by “exon skipping,” which means…
Rare gene mutations in amyotrophic lateral sclerosis (ALS) will be increasingly identified with large-scale, international studies, findings from Project MinE suggest. The study, “Project MinE: study design and pilot analyses of a large-scale whole-genome sequencing study in amyotrophic lateral sclerosis,” was published in the European…
NurOwn, an amyotrophic lateral sclerosis (ALS) treatment candidate, will not be made available under the new Right to Try Act at this time due to a lack of funding alternatives for patients, BrainStorm Cell Therapeutics announced in a press release. The company said it reached this decision after carefully evaluating the Right to…
ProMIS Neurosciences announced that it is moving toward selecting antibodies — from the many identified in earlier work — that specifically target the toxic proteins that accumulate in the brains of amyotrophic lateral sclerosis (ALS) and Alzheimer’s patients, and that may work as therapies. Specifically for ALS, the identified antibodies target TDP43…
Determining genetic profiles from patients’ blood samples can provide a broad view of the biological processes involved in amyotrophic lateral sclerosis (ALS). However, this information may not add valuable diagnostic information because it can’t accurately discriminate ALS cases from conditions that mimic the disease, according to researchers. Their study,…
The noninvasive detection of changes in the blood specific to amyotrophic lateral sclerosis (ALS) may improve the diagnosis of the disease, according to researchers. Their study, “Initial Identification of a Blood-Based Chromosome Conformation Signature for Aiding in the Diagnosis of Amyotrophic Lateral Sclerosis,” appeared in the journal…
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