All these kids are destined to die soon without our help. They have a rare fatal disorder that currently has no treatment. We need to raise $2,000,000 to pay for the medicine that will be used in a clinical trial next year in the United States.
We will need an additional funding to pay for their hospital bills. It will mount to another 1.5-2 MM$.
Our story started when Daniel and his brother Alexander were diagnosed with a rare fatal disease in 2012 and given 10-15 years to live. We started to investigate what could be done to save the boys. A few months later we met Hermance's parents. They had received the same devastating diagnosis for their daughter. We decided to organise 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 with this rare disease who are desperately waiting for treatment. Their time will be cut short if we can't find enough money to pay for their cure. We have already funded all the required studies to start the clinical trial for their treatment. We just need your help to get us over the goal line to save these kids!
Patients? No, just kids
What Is Aspartylglucosaminuria (AGU)?
Aspartylglucosaminuria (AGU) is a rare genetic disease that slows down child development in primary/middle school and often diagnosed as autism. It is one of many lysosomal storage disorders. About 150 children and young adults around the world have this rare genetic trait that also makes them nicest, honest, most helpful, sweetest people ever! However, it also slowly takes their lives away. Up until now, there has been no established cure for the disease. It was first described in the 1960s and yet no cure has been found. The disease is very well described and documented with only one thing left to do: Manufacture the CURE!!!
Gene Replacement Therapy replaces the faulty AGA gene, so the child can produce a missing enzyme. It is a one time treatment. Learn more about the progress and how you can help.
Many AGU patients are currently receiving different chaperone therapies depending on each patient's individual genetic mutation. These therapies can be applied to different diseases.