Steve Bryson, PhD,  science writer—

Significant delays in the diagnosis of amyotrophic lateral sclerosis (ALS) were revealed by a new large-scale analysis of real-world patient records using artificial intelligence (AI). In fact, the median time from the first symptom to an ALS diagnosis was 11 months for these patients. Similar delays were seen regardless…

Researchers have characterized three molecular subtypes of amyotrophic lateral sclerosis (ALS), each one associated with different clinical outcomes, including age of disease onset, disease duration, and survival. These subtypes are based on unique patterns of gene activity in patient brain tissue and were independent of where the onset of…

A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an…

Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721)…

The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…

EpiSwitch, a non-invasive, blood-based test developed by Oxford BioDynamics, successfully stratified patients with amyotrophic lateral sclerosis (ALS) as fast versus slow progressors, according to an interim analysis of the REFINE-ALS study. These findings highlight the potential of the biomarker test to better classify disease progression in ALS patients…

An experimental antisense oligonucleotide that works to suppress the mutant C9orf72 gene — a cause of amyotrophic lateral sclerosis (ALS) — safely lowered the production of damaging proteins and other molecules in a patient in a pilot trial. “While other teams have documented that this gene can be suppressed in cells…

Amyotrophic lateral sclerosis (ALS) can be grouped into four disease subtypes based on patterns of changes in electrical signals in the brain, researchers have discovered. These findings may be valuable to predict future disability and life expectancy, and may help select the patients more likely to benefit in certain…

The U.S. Food and Drug Administration (FDA) has accepted for review Amylyx Pharmaceuticals‘ application seeking approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). The new drug application (NDA) also was granted priority review by the regulatory agency, which reduces review time from the standard…

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…