A vitamin D activator molecule known as CYP27B1 is a biomarker of muscle alterations in patients with amytrophic lateral sclerosis (ALS) and may be a marker of clinical progression of the disease, according to new research. The findings, “The Vitamin D Activator, CYP27B1, is a Novel…
A cancer therapy called imatinib mesylate (sold under the brand name Gleevec) has the potential to slow progression in amyotrophic lateral sclerosis (ALS) and extend survival, research in mice shows. The findings, “Cancer Drug Repurposing for Treating Amyotrophic Lateral Sclerosis (ALS),” were presented at the 2019…
Radicava (edaravone) may prevent functional deterioration in amyotrophic lateral sclerosis (ALS) patients, results from a literature review suggest. Findings from the review, “Safety and Efficacy of Edaravone in Delaying Functional Decline in Amyotrophic Lateral Sclerosis: A Meta-Analysis,” were presented in a poster session at…
The ALS Association has launched a financial aid program, called The Jane Calmes ALS Scholarship Fund, to help students whose lives are affected by amyotrophic lateral sclerosis (ALS) pursue a college degree or vocational certificate. The scholarship program will award $5,000 a year to at least 30…
Biogen’s investigational therapy tofersen can significantly reduce toxic levels of SOD1 protein and may slow disability progression in people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, interim study results show. The positive data from a Phase 1/2 clinical trial support advancing the experimental compound into Phase 3…
Masitinib, the lead experimental medicine being developed by AB Science, can modulate the activity of different cell types known to be involved in inflammation and nerve degeneration in amyotrophic lateral sclerosis (ALS), according to results from a preclinical study. The findings were discussed in a presentation, titled “Post-paralysis treatment with…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Awareness Breeds Compassion. Compassion Drives Enhanced Funding. Funding Generates Heightened Innovative Intervention Ideas. It all starts with awareness. May is ALS Awareness Month. It is not a coincidence that I chose three words beginning with the letter “I.” I believe that a single measure of awareness yields…
Mesenchymal stem cells (MSCs) taken from the bone marrow of patients with amyotrophic lateral sclerosis (ALS) show an ability to control inflammation much like that seen in healthy people, but they respond differently and can be influenced by nearby pro-inflammatory molecules called cytokines, a…
Accumulation of TDP-43 protein is known to drive neurodegeneration associated with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Now, researchers have found that targeting the structure of TDP-43 and blocking its normal activity can halt the death of nerve cells linked to TDP-43 accumulation in ALS and FTD models. The…
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