Tofersen’s Clinical Benefits for ALS Linked to NfL Levels: Trial Data

Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…

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…

Looking back over the course of more than a decade of my husband, Todd, having ALS, I’m struck with how hard we worked to maintain some sense of normalcy, even after he was paralyzed. We joined a Bible study with a small group of people from our church, and we…

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…

I must confess, whenever I’m in a crowded public place, I wear a face mask. Why? Because I’m a person living with ALS and doing everything I can to keep my immune system in tiptop shape. Plus, being the only person wearing a mask doesn’t bother me. In fact,…

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…