The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.
In most people with ALS, and many with FTD, cytoplasmic aggregates of TDP-43 plague degenerating neurons. What causes an RNA-binding protein that spends most of its time in the nucleus to pile up in ...
The TDP-43 RNA-binding protein is found in abnormal cytoplasmic inclusions in affected motor neurons in almost all ALS cases, and mutations in the TDP-43 gene (TARDBP) cause a rare form of familial ...
An 86-year-old woman develops classic amnestic symptoms of Alzheimer’s disease in her last years of life, but upon autopsy, her brain bears but a modest burden of Aβ plaques and tau tangles. Instead, ...