The University California San Diego (UC San Diego) has licensed the right to develop and commercialize an investigational gene therapy for amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, called SynCav1, to CavoGene LifeSciences. The therapy may also benefit people with traumatic brain and spinal cord injuries, and cognitive decline disorders.
Potential Gene Therapy for ALS and Similar Diseases Licensed to CavoGene LifeSciences
ALS life involves a lot of waiting. We wait for people to help us with daily tasks, we wait for medical test results, and we wait for new ALS symptoms. But it’s the constant thoughts of despair and hopelessness that put us in what I call the ALS…
Despite a lack of clinical evidence, complementary and alternative medicines (CAM) are often used by patients with amyotrophic lateral sclerosis (ALS), sometimes in combination with conventional medications, according to a review study. With scarce evidence on the safety and effectiveness of CAM in ALS and the possible risks they…
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…
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…
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