We need your help to fund the 2022 clinical trial for a life-saving gene therapy.
Aspartylglucosaminuria (AGU), is a rare and fatal disease that currently has no treatment. The Rare Trait Hope Fund is dedicated to working towards a cure, but without your help, the kids with AGU will remain destined to die.
There are several fundraising efforts ongoing globally. Donate to help fund the next steps toward a cure.
We need to raise $2,000,000 to pay for the medicine to be used in next year's clinical trial in the United States and $1,500,000 to $2,000,000 to cover hospital bills.
Donate to Help Us Provide a Cure
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
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.
Many AGU patients are receiving chaperone therapies . These therapies can be applied to different diseases.