Current Research

There are researchers, scientists, academic centers, pharmaceutical companies, and advocacy organizations working today to improve the understanding of AGU for a more hopeful tomorrow. Because AGU is so rare, much of the information currently available comes from the experiences shared by individuals living with AGU and from molecular biology research on the function of the AGA enzyme. Research in mice and a natural history study are underway.

How You Can Help the Medical Community
Better Understand AGU

Enrollment for an AGU Natural History Study is Underway

A natural history study is an important step to advance understanding of a disease. A natural history study for AGU is currently enrolling patients. Information on and how to participate in the Natural History Study of Aspartylglucosaminuria (AGU) can be found here.

For more information about a natural history study, please contact:

What is a natural history study?

Why is an AGU natural history study important?

The Commitment of Neurogene to AGU

In addition to sponsoring this educational website, Neurogene Inc. is a biotech company committed to developing life-changing medicines for patients and families affected by rare, devastating neurological diseases.

Neurogene is partnering with researchers and clinicians to gain a greater understanding of AGU in an effort to treat the disease effectively. The latest partnering research can be found here.

AGU Studies

A clinical study entails research involving humans with the intended goal of strengthening medical knowledge. There are two types of clinical studies: non-interventional and clinical trials. Natural history studies are non-interventional, which means that there are no therapeutic interventions. In clinical trials, participants help researchers evaluate the effectiveness, safety, and side effects of investigational therapies that correlate with the disease’s development that may have the possibility of treating a disease.

Studies in AGU are registered on, a website that provides access to information on publicly and privately supported human clinical (treatment) studies and natural history (non-interventional) studies on global diseases and conditions. Information on the Natural History Study of Aspartylglucosaminuria (AGU) can be found here.

Searching “AGU” in the search bar on will result in a list of upcoming trials that are not yet recruiting, trials that are currently enrolling, and trials that have concluded, including any published data resulting from completed studies.