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 studies…
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…
Sometimes we need a nudge to help us get over a mental funk and back into the swing of life. My nudge came from a pair of white ankle-foot orthoses (AFO) with whom I had been having a love-hate relationship. Love ’em or hate ’em, they did end up becoming an…
A specific type of genetic alteration — known as epigenetic changes — observed in patients with amyotrophic lateral sclerosis (ALS) may lead to novel treatments and diagnostic tools, according to a review study. The study, “Epigenetics in amyotrophic lateral sclerosis: a role for histone post-translational modifications…
Scientists created a new prognosis model to predict the risk of respiratory insufficiency or death within six months of follow-up in patients with amyotrophic lateral sclerosis (ALS). The study, “Development of a prognostic model of respiratory insufficiency or death in amyotrophic lateral sclerosis,” was published in the…
On June 21, bicyclists will set out to make a three-day, 270-mile journey to raise funds for the ALS Therapy Development Institute. The Tri-State Trek expedition begins at Boston College in Chestnut Hill, Massachusetts, and ends in Greenwich, Connecticut. Proceeds go to the institute to advance its research into…
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