Topic: amyotrophic lateral sclerosis
In mouse models of ALS, a one-time injection of a short RNA in the spinal cord either prevented the onset of the disease or halted its progression.
Synchron started a clinical trial of its brain-machine interface that will gauge how the technology restores communication in people who are paralyzed.
Rather than targeting gene-gene interactions that kill cells, Maze is going after genetic combinations that stop people from developing deleterious phenotypes.
Disarm Therapeutics signed on Alvin Shih, M.D., the former Retrophin R&D chief who went on to helm Enzyvant, as its full-time CEO.
ArunA Bio CEO Mark Sirgo and Chief Scientific Officer Steve Stice discuss exosomes and how they can be harnessed to treat CNS diseases.
Locana raised $55 million to expand its platform and pipeline for diseases caused by problems in RNA that stop the production of essential proteins.
Cytokinetics’ reldesemtiv failed to beat placebo on a lung function test, but the company thinks a post hoc analysis shows a path forward.
ALS patients on the highest dose of Biogen's tofersen (BIIB067) showed a slowdown in the progression of symptoms in a phase 1 trial.
Neurimmune's antibody targets the misfolded SOD1 protein that has been implicated in a subset of ALS cases.
Sanofi laid out $125 million to take a stake in two of Denali's RIPK1 inhibitors, and now one will be tested in three tough-to-treat brain disorders.