The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sineugene Therapeutics‘ SNUG01, a gene therapy for people with amyotrophic lateral sclerosis (ALS). The designation is given to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…
Living close to water bodies with blue-green algae blooms, or cyanobacteria, is linked to significantly reduced survival among people with amyotrophic lateral sclerosis (ALS), according to a new study. The association was significantly stronger in people who used private well water or participated in water-related activities such as swimming…
Researchers have discovered a pathway in cells that prevents the formation of TDP-43 protein clumps, which contribute to nerve cell damage in nearly all people with amyotrophic lateral sclerosis (ALS). The findings may aid ALS therapies that can activate the pathway and potentially limit TDP-43 clumping and prevent nerve…
An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels…
Edaravone, an approved amyotrophic lateral sclerosis (ALS) therapy sold as Radicava and Radicava ORS, may exert neuroprotective effects by correcting a key molecular feature of ALS, a cell-based study shows. Specifically, edaravone was found to correct the abnormal localization of TDP-43, a protein that is involved in many…
Neighborhood disadvantage, or living in regions with poorer socioeconomic conditions, is significantly associated with shorter survival time among people with amyotrophic lateral sclerosis (ALS), according to a new Michigan-based study. For those living in neighborhoods considered to be among the top 10% of the most disadvantaged, survival was reduced…
Repeated traumatic brain injuries (TBIs), which happen when the brain is damaged by external impacts such as a fall or a car accident, can accelerate the onset and progression of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene, a new mouse study suggests. The effects of…
A machine learning analysis of gene activity in spinal cord samples from people with amyotrophic lateral sclerosis (ALS) revealed new genes associated with the neurodegenerative disorder, a study reports. The newly identified genes included those involved in the function of energy-producing mitochondria, lipid (fat) and iron metabolism, and the…
Constipation, a symptom experienced by about half of amyotrophic lateral sclerosis (ALS) patients, is associated with a significantly faster disease progression and poorer survival, according to a new study. Constipation was also linked to worse anxiety, depression, and sleep problems, data showed. “Objective assessment of these symptoms is critical…
Living next to arable cropland significantly raises the risk of developing amyotrophic lateral sclerosis (ALS), according to a new study in northwestern Italy. A significantly younger age at disease onset also was seen among those living near such farmland, which may include fields of cereals (wheat and barley), beans,…
