News

FUS protein plays a role in the proper functioning and stability of the neuromuscular junction — the site of communication between motor nerves and muscle fibers — and mutations in this protein appear to be toxic to both motor neurons and muscle cells, causing features of amyotrophic lateral sclerosis…

The Phase 2 CENTAUR trial testing AMX0035 for amyotrophic lateral sclerosis (ALS) was designed to include patients with the most severe disease to yield the most powerful results possible, a researcher explained in a recent webinar hosted by Amylyx Pharmaceuticals, which is developing the therapy. The trial…

Scientists have developed a new method to validate antibodies for research, demonstrating its effectiveness by using the protein encoded by the C9ORF72 gene, which, when mutated, may cause amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), a study reports. They also urged its implementation in labs around…

People with amyotrophic lateral sclerosis with a common mutation in the interleukin 6 receptor (IL6R) gene appear likely to have more severe disease symptoms and faster progression than ALS patients without this inherited gene variant, a study reports. This mutation, its researchers reported, is found with a frequency of…

A new muscle science and disease research center has opened at the University of Washington (UW) to investigate better treatment options for disorders such as amyotrophic lateral sclerosis (ALS). The Center for Translational Muscle Research will work in collaboration with the university’s labs and other Seattle-area institutions…

Target ALS joined with the Association for Frontotemporal Degeneration (AFTD) to announce a $5 million grant initiative to spur the development of new therapies and identify biomarkers for both amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD). Project proposals are welcomed from “Industry-Led Consortia (ILCs)” that bring together…

AL-S Pharma AG, a biotech company jointly founded and financed by Neurimmune and TVM Capital Life Science, has enrolled the first patient in its Phase 1 clinical trial assessing the safety and tolerability of AP-101, an experimental therapy for amyotrophic lateral sclerosis (ALS). AP-101 is a human…

Collaborative Medicinal Development (CMD) has enrolled the first patient in its Phase 2/3 clinical trial assessing the safety and efficacy of CuATSM, an investigational therapy for amyotrophic lateral sclerosis (ALS). The trial (NCT04082832) is aimed at evaluating the efficacy and safety of CuATSM. The…

Mexiletine, an oral medicine to treat irregular heartbeats, is safe and effective for reducing muscle cramps in patients with amyotrophic lateral sclerosis (ALS), results from a Phase 4 clinical trial show. To date, this is the only treatment shown to reduce both the number and intensity of cramps…

BrainStorm Cell Therapeutics announced that the Phase 3 clinical trial of its cell therapy candidate NurOwn in 200 amyotrophic lateral sclerosis (ALS) patients is fully enrolled and treatment is underway. The randomized, double-blind and multicenter trial (NCT03280056) is assessing the safety and effectiveness of three administrations of NurOwn into…