Phase 1/2 Trial Testing IONIS-SOD1Rx for ALS Launched After Positive Findings in Animal Models

When ALS Becomes a Hard Act to Swallow

Swallowing. It’s such a simple thing to do! We’re born with the ability to swallow; it happens automatically, and the average person does it approximately 600 times a day. I mean, who actually thinks about swallowing? Well, I do, now that I’ve joined the nearly 80…

Right to Try vs. Right to Say No: An Open Letter to Lawmakers

Dear Congress, The recently enacted federal Right to Try Law (RTT) is really a “Right to Ask” law. And, for the pharmaceutical companies, it’s a “Right to Say No.” Which they certainly almost always will. Case in point: BrainStorm Cell Therapeutics pre-emptively announced that NurOwn, its ALS stem cell treatment candidate,…

Phase 3 Trial of Oral Levosimendan as Respiratory Treatment for ALS Recruiting in US, Europe and Australia

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…

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…

FDA Approves Xeomin for Excessive Drooling, Common in ALS Patients

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…