With your help, we are close to developing a cure for AGU! We are raising money to fund a clinical trial in 2021 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 travel, lodging and meeting fees, drug manufacturing, 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!!!
With the help of other rare disease organizations that have completed the process already, we have come a long way. Here is what we’ve accomplished so far!