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…
Researchers Identify New Genetic Defect Linked to ALS
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…
Caregivers Are Only Human
Ten years ago today, my husband, Todd, was diagnosed with ALS. I’m grateful that our kids have had their dad much longer than we thought they would. When Todd was diagnosed, Sara was 4, and Isaac was just 9 months old, so we thought they might not even remember him.
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.
ALS, Before and After
Author T. H. White‘s collection of novels titled “The Once and Future King” depicts the legend of King Arthur. While the title suggests a symmetrical before and after, in reality, it is rare that such a balance is observed.
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…
This week, I’m celebrating my third anniversary of writing columns for ALS News Today. This special column expresses my gratitude for having the unique opportunity to share my thoughts and wellness knowledge with the ALS community. It is a thank-you to loyal readers for their support, comments, and friendship.
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…
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