News

A CRISPR/Cas9 gene editing system can be used to remove the repeat expansion in the C9ORF72 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), a new study reports. The system showed positive proof-of-concept effects in mouse models and in human cells, reducing the amount of faulty…

ALS News Today brought consistent coverage of the latest research developments and advances in treatment related to amyotrophic lateral sclerosis (ALS) throughout 2022. We look forward to continuing to serve as a resource for the ALS community in the new year. Here, we’ve compiled a list of the…

Patient registries help record how many cases of a particular disease there are across the country, and many also log related genetic, clinical, and biological information. Some U.S. registries, including those dealing with infectious diseases, have mandatory reporting requirements. But for many other diseases, including amyotrophic lateral sclerosis (ALS),…

In amyotrophic lateral sclerosis (ALS), as in many diseases, patient registries, biorepositories, and natural history studies are helpful both in planning clinical trials and as tools for scientists to learn more about how a person’s lifestyle, genetics, and environment can potentially lead to…

Healthcare has long harnessed the power of big data. Examples range from the Human Genome Project, a worldwide 13-year effort to map DNA, to the adoption of electronic medical records— allowing doctors to quickly access patient information at points of care — and the rise of personalized medicine, which tailors…

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…

Health Canada has given a green light to QurAlis‘ request to open a Phase 1 clinical trial of QRL-201, its candidate antisense oligonucleotide (ASO) molecule to protect and repair nerve cells, slowing disease progression, in people with amyotrophic lateral sclerosis (ALS). The global trial, called ANQUR (NCT05633459), will…

BrainStorm Cell Therapeutics has requested a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the agency’s recent refusal to review its application for NurOwn as a treatment for amyotrophic lateral sclerosis (ALS). Type A meetings are scheduled to resolve differences between the…

Health Canada asked more information regarding masitinib as a potential add-on therapy for amyotrophic lateral sclerosis (ALS) before continuing to review a request for its approval. AB Science, masitinib’s developer, now has 90 consecutive days to reply to the notice of deficiency issued by Health Canada, given…

A low dose of aldesleukin, an immunotherapy approved for certain cancers, slowed disease progression and significantly improved survival in people with amyotrophic lateral sclerosis (ALS), particularly in those with less aggressive disease. These are the most recent results from the MIROCALS Phase 2 trial (NCT03039673), which tested aldesleukin…