RNA molecules that are associated with genetic disorders characterized by damage to nerve fibers are found at higher-than-usual numbers in compartments of nerve cells, a new study reports. This finding may lead to new ways of treating neuromuscular diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy and…
mRNA Linked to ALS and Other Disorders Found at High Levels in Nerve Fibers, Study Reports
Swiss biopharma GeNeuro has secured the exclusive worldwide license to the clinical development program for a pHERV-K Env antibody after preclinical results showed its potential to treat amyotrophic lateral sclerosis (ALS). The company exercised its option for the rights to the investigational therapy under the terms of a January 2017…
Voyager Therapeutics’ experimental therapy VY-SOD102 shows promise for treating patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results in animal models. The preclinical data were presented at the Congress of the European Society of Gene and Cell Therapy…
My (Ice) Bucket List
“… And up through the ground come a bubblin’ crude.” Like Jed Clampett of “Beverly Hillbillies” fame, the ALS Association in 2014 found itself in the unexpected circumstance of commanding assets beyond its wildest dreams. The national display of generosity, hope, and compassion dubbed the ice…
Tiglutik, an oral suspension of riluzole, is now available as a treatment for amyotrophic lateral sclerosis (ALS) in the U.S., ITF Pharma, the therapy’s developer and a subsidiary of Italfarmaco, recently announced. Rilutek (riluzole, marketed by Sanofi), has been available in the U.S. as 50 mg tablets…
The Art of ALS Adaptability
Life with ALS often feels like a crash course in adaptability but being adaptable doesn’t come naturally for many of us. However, whether you’re the patient, caregiver, or a family member, I believe you can learn how. Read on and let’s get started. In my pre-ALS life…
SOD1 gene mutations can contribute differently to the degenerative process of nerve cells involved in the progression of amyotrophic lateral sclerosis (ALS), newly developed worm models of the disease reveal. This finding was reported in the study, “Single copy/knock-in models…
One of the most awful parts of my diagnosis was the loss of my career. Teaching was more than a job to me — it was part of my identity. I wish I knew then that my loss wouldn’t be as total as I had imagined. Don’t give…
Chip technology — much like an organ-on-a-chip that can simulate the workings of tissue or organs — was used to recreate amyotrophic lateral sclerosis (ALS), allowing researchers to identify two treatments for blood cancers — rapamycin plus bosutinib — as a possible ALS combination therapy. The study, “Microphysiological 3D model…
Researchers have discovered that mast cells and neutrophils — two types of immune cells — are involved in the degeneration of peripheral motor nerve cells and progression of amyotrophic lateral sclerosis (ALS). These findings also clarify why masitinib — an investigational therapy for ALS that targets mast cells…
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