Sangamo Therapeutics and Pfizer will work together on gene therapies they hope will overcome mutations that cause ALS and frontotemporal lobar degeneration. Gene therapy works by delivering a healthy copy of a malfunctioning gene to a patient. The therapy that Sangamo is developing replaces a faulty C9orf72 gene. It consists…
Cell stress, such as a viral infection, may trigger the production of faulty proteins from the C9orf72 gene, a leading contributor of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), say researchers at the University of Michigan. Their study, “RAN translation at C9orf72-associated repeat expansions is selectively enhanced…
Rural residents with high school diplomas are at lower risk of developing dementia and cognitive impairment than those with less education, a study indicates. The study has healthcare policy implications, including providing more care to those in rural areas with dementia and cognition-related disorders such as Alzheimer’s and ALS. Researchers…
Throughout 2017, ALS News Today brought you daily coverage of amyotrophic lateral sclerosis (ALS)-related advocacy events, clinical studies, and the latest research updates. We have compiled a top 10 list of the most-read ALS news stories of that year, with a brief description of what made them interesting and…
Gene editing may be a viable tool to slow disease-causing processes in amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, a mouse study indicates. Published in the journal Science Advances, the study demonstrated that the method delayed disease…
Q Therapeutics of the United States and REPROCELL of Japan are teaming up to develop stem cell-based therapies for central nervous system diseases such as ALS and transverse myelitis, or TM. The collaboration will meld Q Therapeutics’ expertise in nerve cell therapy with REPROCELL’s expertise in stem cell treatments.
Patients with amyotrophic lateral sclerosis (ALS) experience a gradual loss of the ability to walk, swallow and even breathe, and there’s no cure. It’s incredibly important that patients seek specialized treatment to address the different aspects of the disease. MORE: Find out seven ways to diagnose ALS In…
Two young companies, QurAlis and Kernal Biologics, are winners of the Amgen Golden Tickets for innovative bioscience projects — in this case, work that might lead to better treatments in amyotrophic lateral sclerosis (ALS) and cancer immunotherapy, Amgen and LabCentral announced. QurAlis is developing therapies for three different forms of…
The Wave Life Sciences therapy WVE-3972-01 reduced markers of the form of ALS that scientists believe stems from a gene mutation, studies show. Researchers said the potential treatment blocked faulty versions of messenger RNA associated with the production of abnormal C9orf72 protein. The RNA is an intermediary between the C9orf72 gene…
MediciNova announced successful top-line results from a Phase 2 clinical trial of MN-166 (ibudilast) as an adjunct therapy to Rilutek (riluzole) in patients with amyotrophic lateral sclerosis (ALS). The treatment was safe and well-tolerated – meeting the trial’s primary endpoint – and was found to be more effective at…
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