Alzheimer’s disease starts with a sticky protein called amyloid beta that builds up into plaques in the brain, setting off a chain of events that results in brain atrophy and cognitive decline.
Updated: BioMarin’s hemophilia A gene therapy Roctavian wins FDA approval after delay and rejection, priced at $2.9M
The FDA approved the first gene therapy to treat the genetic bleeding disorder hemophilia A on Thursday, capping a turbulent regulatory saga for BioMarin, which
June 30, 2023