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,…
Right to Try vs. Right to Say No: An Open Letter to Lawmakers
Treatment of amyotrophic lateral sclerosis (ALS) with investigational compound ibudilast (MN-166) as an add-on to Rilutek (riluzole) improves patients’ functional activity, quality of life, and muscle strength, data from MediciNova’s Phase 2 trial show. MediciNova is now recruiting participants for a Phase 1/2 biomarker trial (…
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…
For most of my adult life, keeping track of my health was easy peasy — at least, it was before I had ALS. I always followed the recommended guidelines for annual exams, participated in my employer’s work site wellness screens and made sure my immunizations were up…
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…
Over the past year, I have become an expert in pain — something I never wanted to be. However, daily severe muscle spasms, cramping, one surgery, and an emergency room visit later, here I am. Despite my plethora of pain medications, I still suffered. Then, I realized that…
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…
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