Mutations in the gene NEK1, a major genetic cause of amyotrophic lateral sclerosis (ALS), may work to drive the disease by destabilizing the structure of nerve fibers and disrupting the movement of molecules inside nerve cells, according to a new study. The findings, which show for the first time how…
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Note: This story was updated Aug. 22, 2023, to correct there are four ALS approved therapies widely available in the U.S. Diarrhea associated with a common amyotrophic lateral sclerosis (ALS) treatment could be a substantial barrier to complying with the medication, according to surveys by Napo Pharmaceuticals. While…
Elevated blood levels of the Cystatin C protein are significantly associated with faster disease progression and shorter survival in people with amyotrophic lateral sclerosis (ALS), according to a study in China. The protein may serve as a biomarker to predict disease outcomes in people with ALS and identify those…
People with amyotrophic lateral sclerosis (ALS) who have a more active innate immune system — the part of the immune system that acts as a first responder to threats — tend to have faster disease progression and poorer survival, a study of large data showed. Findings suggest that measuring…
Early treatment with Radicava (edaravone), an approved therapy for amyotrophic lateral sclerosis (ALS), significantly reduced the risk of hospitalization, need for ventilatory support, or death compared with delaying treatment for six months. That’s according to analyses of data from Study 19 (NCT01492686), the Phase 3 clinical trial…
Mitsubishi Tanabe Pharma Canada (MTP-CA) is teaming up with the Canadian Neuromuscular Disease Registry (CNDR) in Canada’s first real-world, evidence-generating study of Radicava (edaravone) in amyotrophic lateral sclerosis (ALS). Data are expected to provide real-life insights into the survival benefits of Radicava in the progressive neurodegenerative…
Increased accumulation of p62, a protein involved in a cellular recycling process called autophagy, in the spinal cord is associated with worse survival outcomes in people with sporadic amyotrophic lateral sclerosis (ALS), a study revealed. Autophagy is the cellular process responsible for breaking down proteins and other complex molecules…
Coya Therapeutics is planning to file a request with the U.S. Food and Drug Administration (FDA) to continue testing its investigational therapy COYA 302 for the treatment of amyotrophic lateral sclerosis (ALS) in a Phase 2 clinical trial. The company is preparing a meeting with the regulatory agency…
Researchers have identified new genetic variants that might influence survival among sporadic amyotrophic lateral sclerosis (ALS) patients in Japan. Genetic analyses in more than 1,000 ALS patients uncovered that small changes in the FGF1, THSD7A, and LRP1 genes could affect patients’ prognosis. In patient-derived nerve cells, the identified variants led…
The axeALS Foundation is inviting people in the U.S. with amyotrophic lateral sclerosis (ALS) and demonstrable financial needs to apply for a grant to defray the cost of living with the progressive neurodegenerative disease. The application deadline is midnight Aug. 15. Institutions may apply as well, although…
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