Marisa Wexler, MS,  senior science writer—

Four experimental treatments for amyotrophic lateral sclerosis (ALS) have been tested in the HEALEY ALS platform trial and two, CNM-Au8 and pridopidine, have shown promising results and are moving toward Phase 3 testing. The others, zilucoplan and verdiperstat, failed to show any benefit and are no…

Sleep problems may become evident in people with amyotrophic lateral sclerosis (ALS) several years before the characteristic motor symptoms of the disease develop, a new study shows. The study suggests alterations in a brain region called the hypothalamus, which are seen in early disease stages and sometimes even…

Oral therapy IFB-088 (icerguastat) was safe and significantly slowed disease progression in certain people with amyotrophic lateral sclerosis (ALS), according to new data from a Phase 2 clinical trial. “We are very encouraged by the results we observed with IFB-088,” Pierre Miniou, CEO of IFB-088’s developer Inflectis Bioscience,…

uniQure is planning to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162, its one-time gene therapy for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The open-label Phase 1/2 study, called EPISOD1 (NCT06100276) is intended to…

People with diabetes or high cholesterol may be less likely to develop amyotrophic lateral sclerosis (ALS) or other motor neuron diseases (MNDs), but heart and metabolism problems are linked with faster disease progression and worse survival rates among people who already have MND. That’s according to the study, “…

People with amyotrophic lateral sclerosis (ALS) who get a feeding tube placed after losing a lot of weight are more likely to die within a few months than those who receive feeding support before major weight loss, a study reports. Findings suggest that feeding tubes placed before substantial weight…

The HEALEY ALS platform trial, which is simultaneously testing multiple potential treatments for amyotrophic lateral sclerosis (ALS), is amending its master protocol to allow a longer follow-up time and collection of blood cells for use in future research. Slight modifications to the enrollment criteria and visit schedule will…

The U.S. Food and Drug Administration (FDA) signaled that it may be willing to consider granting accelerated approval to CNM-Au8 to treat amyotrophic lateral sclerosis (ALS), based on data from clinical trials and ongoing expanded access programs. The therapy’s developer, Clene, said it will put together an…

People with mutations in the C9ORF72 gene who develop amyotrophic lateral sclerosis (ALS) show signs of damage to certain brain regions years before the appearance of disease symptoms, a study reports. The findings suggest that looking for changes in brain structures could help predict which people with C9ORF72 mutations…

Long-term treatment with the cell therapy NurOwn (debamestrocel) was found to significantly extend survival in people with amyotrophic lateral sclerosis (ALS) when compared with a matched control group from previous clinical trials, according to new data. That data, from an expanded access program (EAP), was shared by NurOwn’s…