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…
Continuing its support for a potential therapy for amyotrophic lateral sclerosis (ALS), Australia-based organization FightMND has granted about $700,000 to biopharmaceutical company Collaborative Medicinal Development. The money will fund a randomized, double-blind, placebo-controlled study of CuATSM, one of CMD’s lead investigational therapies. According to a news release, the…
Newly diagnosed amyotrophic lateral sclerosis (ALS) patients often experience poor sleep quality due to symptoms of depression and difficulty turning in bed, a patient survey shows. The study, “Poor Sleep Quality in Patients With Amyotrophic Lateral Sclerosis at the Time of Diagnosis,” was published in the Journal…
Project ALS and Columbia University are launching a new platform with a goal of discovering more useful treatments for amyotrophic lateral sclerosis (ALS) patients, and pushing the most promising candidates to clinical trials. Core, as it…
Aquestive’s new drug application for Exservan — an investigational oral film of riluzole to be used as an add-on therapy for amyotrophic lateral sclerosis (ALS) — has been accepted by the U.S. Food…
The U.S. Food and Drug Administration approved MediciNova’s plans for a pivotal Phase 2b/3 clinical trial that will explore the potential of ibudilast (MN-166) in patients with amyotrophic lateral sclerosis (ALS). Pending the success of the trial, the company hopes the collected clinical data will support the…
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