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 A protein responsible for speeding the rate of chemical reaction in the cell of the living organism. Enzymes support a wide range of functions in an organism, as they spark chemical reactions. Enzymes work with, or interact with, substrates (the underlying surface or substance). When reacted together, the enzyme and substrate result in a new product or molecule that then separates from the original enzyme and goes on to create new reactions in the cell.. Research in mice and a There are two types of natural history studies: prospective and retrospective. A natural history study examines a group of people who have a specific medical condition or disease or are at risk of developing one. A natural history is non-interventional and collects health information in order to understand how the medical condition or disease develops and how to possibly treat it. No treatment or investigational product is given. In a prospective study, this data collection is forward-looking and is done over time in the future. In a retrospective study, researchers review and examine factors related to an outcome in the past by looking back on past exposures and medical events. 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 A rare neurodegenerative lysosomal storage disease (LSD) characterized by developmental delay, psychomotor regression, worsening intellectual disability, gait disturbance, and shortened lifespan. There are currently no available treatments for AGU. (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.
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 A study conducted in humans that evaluates the effectiveness, safety, and side effects of investigational products (which could include both medications, treatments, or devices) that have the possibility of treating a disease in that particular patient group.. 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 ClinicalTrials.gov, 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 ClinicalTrials.gov 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.