News

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…

A Harvard Medical School professor and scientist, Sabrina Paganoni, has won the 2019 American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) Scientific Impact Award for her work in a Phase 2a trial testing fingolimod as a potential treatment for amyotrophic lateral sclerosis (ALS). Fingolimod is marketed as Gilenya (by Novartis), an…

The final amyotrophic lateral sclerosis (ALS) patient treated with oral AMX0035 in the CENTAUR study has completed all scheduled follow-up visits and researchers will now evaluate data collected on the potential treatment’s safety and efficacy, Amylyx Pharmaceuticals, its developer, announced. Final results of the Phase 2 study (NCT03127514), which…

Experimental treatment reldesemtiv appears to reduce functional deterioration in patients with fast progressing amyotrophic lateral sclerosis (ALS), a new analysis of the FORTITUDE-ALS trial shows. The additional analyses were presented by the trial’s principal investigator, Jeremy M. Shefner, MD, PhD, a professor at the Barrow Neurological Institute…

Mutations in the annexin A11 gene (ANXA11), seen in familial amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, disrupt the transport of key RNA molecules inside neurons, recent research shows. The annexin A11 protein is a molecular adaptor required for RNA molecules, essential for neurons’ function, to hitchhike on lysosomes…