Rare Disease Facts
AGU is one of many rare diseases. Term "rare disease" means that only a small number of population, less than 200,000 in USA, is affected by a particular disease. Also, 
  • There are approximately 7,000 different types of rare diseases and disorders, with more being discovered each day; 
  • 30 million people in the United States are living with rare diseases. This equates to 1 in 10 Americans or 10% of the U.S. Population; 
  • If all of the people with rare diseases lived in one country, it would be the world’s 3rd most populous country; 
  • 80% of rare diseases are genetic in origin, and thus are present throughout a person’s life, even if symptoms do not immediately appear; 
  • According to the Kakkis EveryLife Foundation, 95% of rare diseases have not one single FDA approved drug treatment; 
  • Approximately 50% of rare diseases do not have a disease specific foundation supporting or researching their rare disease. 
With your help, we are set to develop a cure for one of the rare diseases, AGU. We are currently funding pre-clinical treatment development work in several Universities in US and Europe. There are several fundraising events you can participate in and support funding for this rare disease.
Following FDA drug approval process (fully described here), the first step for a new treatment approval is to obtain "Investigational New Drug" designation from FDA that allows to test a New Drug diagnostic or therapeutic potential in humans. For that, we (drug's sponsor) have to complete the screening of the new drug for pharmacological activity and acute toxicity potential in animals.
With the goal in mind to develop a treatment for AGU and with the help of those who went through the same process already, here is what we've done so far.