We need your help paying for the life-saving drug !
Aspartylglucosaminuria (AGU), is a rare and fatal disease that currently has no treatment. Rare Trait Hope Fund is dedicated to find a treatment for this disease, but without your help, the kids with AGU are destined to die.
Total cost of drug manufacturing and testing is US$ 5,824,600. We already paid over 70% . We need your help to pay off the drug and give it to AGU kids.
Please donate directly on the website or through GoFundMe in the United States, Canada, Europe, or Australia.
Aspartylglucosaminuria (AGU) Patient Registry and Natural History Study has been launched. Patients can share their AGU story and play a pivotal role in advancing research and developing new treatments. Patient can join here to be part of the journey.
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After years of efforts, all pre-clinical work and studies are completed. Every vendor (but one!) had submitted their reports and the team working hard to finalize everything required to submit the IND (Initial New Drug) Application to the Food and Drug Administration (FDA) to start the clinical trial.
In 2012, brothers Daniel and Alexander were diagnosed with a rare fatal disease called Aspartylglucosaminuria (AGU) and given 10-15 years to live. We began to investigate what could be done to save the boys.
A few months later we met the parents of Hermance, who had received the same devastating diagnosis. We collaborated to organize and fund the research community that would develop the treatment for this disease.
Since then, we've met parents from around the world who are fighting to save the lives of their children. Below are the stories of some of the kids who are desperately waiting for a treatment. Their time will be cut short if we can't raise enough money to deliver their cure.
We have funded all the required studies to start the clinical trial to treat the kids with AGU. We just need your help to get us over the goal line to save these kids!
Aspartylglucosaminuria (AGU) is a rare genetic lysosomal disorder that slows developmental progress in childhood. In primary and middle school, children with AGU are often labeled as having autism spectrum disorder or general developmental delay. Unlike typical autism, however, AGU causes additional health problems and is progressively degenerative, with symptoms that worsen over time.
Fewer than 300 children and young adults worldwide live with AGU today. They are often noted for their kindness, honesty, and eagerness to help—qualities that shine even as the disease gradually diminishes their abilities and shortens their lives.
Since AGU was first described in the 1960s, researchers have mapped its genetic cause and conducted a lot of research, but an effective treatment has remained out of reach. For over a decade, we’ve been dedicated to changing that—advancing a gene-therapy program aimed at transforming this hard-won knowledge into the first life-altering therapy for the entire AGU community.
What is a gene? What does it do in a human body? Are changes in genes good or bad? What is gene therapy? Can it help patients?
Many AGU patients are receiving chaperone therapies . These therapies can be applied to different diseases.
Symptoms of AGU aka Aspartylglucosaminuria. Does my child have AGU? How to get tested? Is the treatment available?
Information about AGU; differential diagnosis, disease progression, available testing. Disease natural history study, etc.