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 students whose…
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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.
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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Studying Epigenetic Changes Could Lead to New ALS Treatments and Biomarkers, Researchers Suggest
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…
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New Prognosis Model Predicts Risk of Respiratory Insufficiency or Death in ALS Patients, Study Says
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…
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…
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