Steve Bryson, PhD, science writer —

A European Medicines Agency (EMA) committee again has delayed issuing an opinion on whether to grant conditional approval to masitinib, a potential add-on oral therapy for amyotrophic lateral sclerosis (ALS). AB Science, the therapy’s developer, now expects a recommendation in the second quarter of this year. The…

The proportion of people diagnosed with familial amyotrophic lateral sclerosis (ALS) among all cases is 8%, according to a pooled analysis of studies published worldwide. Still, data show there was a wide variation of estimates across the studies based partly on geographical differences, study design, the definition of familial…

A previously unknown mutation in the SPTLC2 gene was identified in two patients who developed juvenile-onset amyotrophic lateral sclerosis (ALS), a study reports. The mutation significantly increased the production of certain types of fat-like molecules called sphingolipids, resulting in early-onset muscle weakness, progressive motor impairment, and involuntary tongue movements.

Throughout 2023, ALS News Today brought you daily coverage of the latest clinical research and scientific breakthroughs related to amyotrophic lateral sclerosis (ALS). Here are the year’s top 10 most-read articles, each with a brief description. We’re excited to remain a dependable resource for the ALS community in…

Early data from Phase 2 programs showing that CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS), induced significant reductions in a nerve damage biomarker but did not meet the criteria for accelerated approval at this time, the U.S. Food and Drug Administration (FDA) concluded. Despite the…

Treatment with Relyvrio (sodium phenylbutyrate and taurursodiol) significantly reduced blood levels of neuroinflammatory biomarkers in people with amyotrophic lateral sclerosis (ALS) as early as three months, according to a post hoc analysis of the CENTAUR trial. Data from the Phase 2 study (NCT03127514) demonstrated that reductions in…

Toxic compounds produced by cyanobacteria, which have been identified as risk factors for developing amyotrophic lateral sclerosis (ALS), were detected in air and earth samples collected around the Great Salt Lake in Utah, a study reports. “An understanding of the types and concentrations of cyanotoxins present in air is…

SOL-257, an experimental one-time gene therapy, was well tolerated and significantly improved disease outcomes in two distinct mouse models of amyotrophic lateral sclerosis (ALS). Developed by Sola Biosciences, SOL-257 is designed to clear the toxic TDP-43 protein that accumulates and forms clumps in ALS nerve cells, contributing to…

Pregnancy can worsen the progression of amyotrophic lateral sclerosis (ALS), regardless of whether symptom onset occurs before or during pregnancy, according to a review study. Still, most of the cases reviewed showed that ALS did not affect a woman’s ability to give birth, naturally or by cesarean section, or…

People with amyotrophic lateral sclerosis (ALS) due to mutations in the FUS gene may benefit from interferon-gamma treatment to delay disease onset and/or progression, an early preclinical study suggests. Researchers found that an immune signaling protein called interferon (INF)-gamma protected patient-derived motor neurons from oxidative stress. INF-gamma also restored…