With your help, we are close to a treatment for AGU! We are raising money to fund an upcoming clinical trial in the United States which will utilize a benign virus to deliver a new AGA enzyme to AGU kids and disperse it throughout their brains. Click here to see how it works!
There are a lot of steps involved getting a treatment to clinical trial. We are in the process of finalizing the location of the clinical trial and identifying and retaining a project manager with research experience to manage the Investigational New Drug process with the FDA and establish the treatment protocol. The money we are raising will be used to cover expenses associated with the preparation for and conduct of the clinical trial. These include drug manufacturing, travel, lodging and meeting fees, testing (safety, biodistribution and kinetics), clinical fees, hospital fees, clinical research organization costs (data processing) and related costs. It sounds like a lot, but we are actually SO CLOSE!!!