Continuous monitoring of certain biomarkers in people with amyotrophic lateral sclerosis (ALS) may inform about the risk of disease progression and the response to edaravone, according to interim data from the ongoing REFINE-ALS biomarker study. Mitsubishi Tanabe Pharma America (MTPA), the developer of edaravone, recently shared these findings…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Stealth Biotherapeutics’ investigational therapy SBT-272 for the treatment of amyotrophic lateral sclerosis (ALS). Aimed at speeding the development of new treatments for rare, life-threatening diseases, orphan drug status offers regulatory support and certain financial incentives…
Four years after its discovery via an artificial intelligence (AI) platform, Verge Genomics will test its amyotrophic lateral sclerosis (ALS) treatment candidate VRG50635 — a PIKfyve inhibitor — in a first-in-human trial. The Phase 1 clinical trial (ISRCTN14792372), which is now enrolling participants, is being conducted at the…
CuATSM, a potential amyotrophic lateral sclerosis (ALS) therapy that’s now in clinical trials, may work by altering how certain cells in the brain generate energy, a new study suggests. Researchers say these findings may help to identify patients most likely to respond to the experimental treatment. “Screening patient derived…
Proposed updates to the National ALS Registry’s case-identification algorithm and classifications may help provide a better estimate of the prevalence of amyotrophic lateral sclerosis (ALS) in the U.S., a study reported. Under the revision, individuals previously classified as “possible ALS” cases and excluded from the registry would now…
Higher long-term exposure to certain forms of air pollution — specifically coarse particulate matter, such as that from traffic pollutants — significantly increases the risk of amyotrophic lateral sclerosis (ALS) in postmenopausal women, a study showed. “Although the effects noted within the current study are limited by sample size…
High total protein levels in the cerebrospinal fluid (CSF) — the liquid that surrounds the brain and spinal cord — are linked to shorter survival among amyotrophic lateral sclerosis (ALS) patients with limb-onset disease, a study showed. The researchers noted a similar association was found for high CSF…
Deep machine-learning models using neural networks — computer algorithms inspired in the biological networks of the human brain —may help to predict the course of amyotrophic lateral sclerosis (ALS), a new study reports. The models also consistently showed that a longer delay between disease onset and diagnosis is a…
Starting treatment with riluzole earlier in the disease course could prolong overall survival for people with amyotrophic lateral sclerosis (ALS), a database study suggests. A one-year delay in beginning with riluzole may lower median survival by 1.9 months and a two-year delay by a median of 4.9 months, its…
The U.S. Food and Drug Administration (FDA) is funding several novel research projects related to amyotrophic lateral sclerosis (ALS) and other rare neurological disorders through its orphan drug grants program. In total, the regulatory agency has awarded 19 new grants and two contracts, providing more than $38 million…
