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Above: kids with Aspartylglucosaminuria
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.
WHAT'S HAPPENING AT RARE TRAIT
WHAT'S HAPPENING AT RARE TRAIT
AGU NHS
AGU NHS
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.
Rare Rhythms
Rare Rhythms
Join us for an unforgettable evening of history, music, and momentum as we gather to celebrate and recognize the urgency of ultra-rare diseases and the dedication of those working to find treatments for them.
Clinical trial
Clinical trial
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 IND (Initial New Drug) Application to the FDA.
Our Beginnings
Our Beginnings
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!
Patients? No, just kids
Patients? No, just kids
What is Aspartylglucosaminuria?
What is Aspartylglucosaminuria?
Aspartylglucosaminuria (AGU) is a rare genetic lysosomal disorder, that slows development in childhood. In primary and middle school, kids with AGU are often diagnosed with Autism. What differentiates AGU from typical cases of Autism are its range of other negative health effects and the fact that it's severity worsens over time.
Globally, about 150 children and young adults have Aspartylglucosaminuria (AGU). AGU produces some of the most kind, sweet, honest, and helpful people ever! Unfortunately, it also slowly takes their lives away.
AGU has been well-described and documented since its discovery in the 1960's, but to this day there is no established cure. The final step is to MANUFACTURE THE CURE.
Curing Aspartylglucosaminuria
Curing Aspartylglucosaminuria
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.
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