Showing 2696 results for "amyotrophic lateral sclerosis (ALS)"

Single Injection of RNA Therapy Blocks Degeneration, Prevents Disease Progression in Mice Models of Familial ALS

A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…

NPT520-34

NPT520-34 is a new small molecule oral treatment being developed by Neuropore Therapies for potential use against neurodegenerative disorders like amyotrophic lateral sclerosis (ALS) and Parkinson’s disease. The therapy is believed to reduce neuroinflammation in the brain. What is ALS? ALS is a progressive disease…

Ultomiris (Ravulizumab-cwvz)

Ultomiris (ravulizumab-cwvz) is an antibody-based medication that was being developed by Alexion Pharmaceuticals as a potential therapy for amyotrophic lateral sclerosis (ALS). However, after a Phase 3 clinical trial indicated that Ultomiris was not effective in treating ALS, Alexion in 2021 discontinued its development for the…

NT0502

NT0502 is an experimental oral treatment being developed by Neos Therapeutics to reduce excessive salivation — drooling — a common problem in patients with amyotrophic lateral sclerosis (ALS). What is ALS? ALS is a neurological disease characterized by the progressive damage and loss of motor neurons, which are…