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Natural History Study


Although, clinical trial in humans is a few years away, the disease Natural History Study (NHS) is ongoing. To evaluate an experimental treatment and determine if it has slowed, stopped, sped, reversed or had no impact on disease progression, FDA has to have an untreated disease progression data for any particular disease.

A lot of patient data is available for the Finish major mutation, which is not a case for all the other AGA gene mutations. Because there are so few known patients with different AGU mutations, it is very important that parents/guardians seriously consider having their child participate in the AGU Natural History Study. The purpose of this study is to track the untreated course of the disease, with the goal of arriving at clinical outcome measures. If you are unable to travel and wish to participate in the NHS, your child’s medical record and any other test results are all valuable to the natural history study. We appreciate your help. 

To learn more about the NHS study or to get help how to send medical record information, please contact us.