Mutations in the UBQLN2 gene, known to cause amyotrophic lateral sclerosis (ALS), promote the buildup of toxic waste in brain cells by preventing the normal function of two cellular degradation mechanisms, a study has found. In addition to its known role in the proteasome, a mechanism used…
News
Denali Therapeutics and Sanofi announced a pause in clinical testing of their small molecule inhibitor DNL747 in favor of work on a possibly more effective compound, DNL788, for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s, and multiple sclerosis. DNL747 was seen to be safe…
Genetic variants of the NEK1 gene that cause the coded protein to lose its function seem to increase the odds of developing amyotrophic lateral sclerosis (ALS) by more than ninefold, with these patients significantly more likely to experience weakness in their hands as a first symptom, a study has…
The ALS Association and I AM ALS have awarded BrainStorm Cell Therapeutics $500,000 to support an amyotrophic lateral sclerosis (ALS) biomarker study based on the biotechnology company’s pivotal trial into its NurOwn therapy. Specifically, the combined grant — $400,000 is from the ALS Association —…
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.
A cancer treatment called tamoxifen, which previously showed promise in preclinical studies for the treatment of amyotrophic lateral sclerosis (ALS), provides only modest and short-lasting benefits to patients with the disease, a small Phase 1/2 clinical trial has found. According to the study’s researchers, larger studies and longer…
Treatment of people whose symptoms are suggestive of amyotrophic lateral sclerosis (ALS) but likely linked to a retrovirus called human T-cell lymphotropic virus type I (HTLV-I) should focus on alleviating symptoms and lowering the viral load, a case report highlights. The report, “Human T-cell Lymphotropic Virus…
The teeth of people with amyotrophic lateral sclerosis (ALS) show their bodies metabolize metals — from chromium to zinc — in abnormal ways starting in infancy, and these changes predispose them to ALS disease as adults. The study, which looked at how metal uptake early in life can affect…
The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced. “BrainStorm made…
Statins, often prescribed to lower cholesterol levels, are not associated with poorer overall survival in people with amyotrophic lateral sclerosis (ALS) and should not be discontinued on that basis, a German study has found. The study, “Statins, diabetes mellitus, and prognosis of Amyotrophic…
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