News

Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen’s tofersen, now named Qalsody, for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The decision, which marks the first conditional approval for ALS in the country, comes about eight…

A new technology powered by artificial intelligence (AI) may one day help doctors diagnose people in the early stages of amyotrophic lateral sclerosis (ALS), according to data from a clinical trial. Developed by Pison, the technology was able to detect involuntary muscle twitches, or fasciculations, and changes in…

Arrowhead Pharmaceuticals will seek permission later this year for the first clinical trial of ARO-SOD1, its RNA-based therapy for amyotrophic lateral sclerosis (ALS), according to a company press release. Further details about Arrowhead’s plans for testing its investigational RNA therapy are expected at a company Research and…

An immune protein that is mostly produced in neurons was found to drive damage to mitochondria, which produce energy for cells, and nerve fiber loss, a study has found. Suppressing the protein, called gasdermin-E (GSDME), prevented nerve fiber loss in cells derived from people with amyotrophic lateral sclerosis (ALS),…

Cold temperatures can activate a complex called the proteasome that cells use to clear themselves of unneeded proteins, including the protein clumps associated with amyotrophic lateral sclerosis (ALS), a new study reports. In cell models of the disease, exposure to cold temperatures for a prolonged time significantly lowered the…

A certified care center of excellence for amyotrophic lateral sclerosis (ALS) has opened in Hackensack Meridian Neuroscience Institute at the Jersey Shore University Medical Center. The ALS Center is part of the medical center’s Dr. Robert H. Harris Neuroscience Treatment Center in Neptune, New Jersey. “Our multidisciplinary…

A first patient has been dosed in a Phase 1 clinical trial evaluating QRL-201, an experimental antisense oligonucleotide (ASO) molecule in treating amyotrophic lateral sclerosis (ALS), the therapy’s developer, QurAlis, announced. Dubbed ANQUR (NCT05633459), the study is reported to be the first into a potential therapy…

Transplants using mesenchymal stromal cells or MSCs — a type of stem cell found in bone marrow — delivered into the spinal canal can significantly extend survival in people with amyotrophic lateral sclerosis (ALS) compared with what would be expected based on their clinical characteristics,…

AI Therapeutics‘ experimental therapy AIT-101 led to reductions in levels of toxic proteins in people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, according to data from a clinical trial. The Phase 2a clinical trial also met its main goals of showing that AIT-101…