Background


Observing mine and other kids throughout many years, I came to believe that my sons' delays were not all I've been told; there was something else standing between my kids and their ability to become what they want to be.
 Daniel was born in 1994 as a beautiful, perfect, and healthy baby. When he was three years old, we accepted that he was somewhat behind other kids. It was nothing that we couldn't handle at that time, because he would soon catch up, as we were told. We had a great support at school, community and private therapists. He worked very hard to become a normal kid. We started noticing the same problems with Alexander when he was a little over one year old. From that moment on, we knew that our boys did not have just allergies, infections, low muscle tone, diarrhea, short attention span, etc. It was something else that was taking our kids life away from them, and taking them from us. We started the search for the answers! 

When we finally learned the correct diagnosis in 2012, it devastated us. It took us 16 years to learn that our oldest son Daniel has Aspartylglucosaminuria (AGU), a rare fatal disorder, and a month later we got confirmation that our younger son Alexander has AGU also. During sixteen years spent looking for an answer, we never had a doubt that when we found it, we could cure our boys and give them a chance for a normal life. Unfortunately, when finally we knew the problem, we could not help them! 

The inspiration came from Alexander, our intuitive little Spiderman. One day he said: “Mommy, don’t cry. Let’s watch a movie!“. Of course it was a Spiderman movie, the one that ends with the phrase:”With great power comes great responsibility.” After the movie Alexander asked me: ”Why responsibility comes with power?” At that instant I knew the answer! Because I have the power of knowing what’s wrong with the kids, now I have a great responsibility for these two human beings and all the others to find the cure! 

Aspartylglucosaminuria (AGU) is a rare genetic disease that slows down child development in the primary/middle school. It belongs to a lysosomal storage disorders. There are just over 300 children and young adults around the world that got a rare genetic trait that makes the nicest, honest, most helpful and the sweetest people ever. It also slowly takes their lives away at the same time. Up till now, there is no established cure for the disease. It has been first described in 1960s and no cure has been found so far. The disease is very well described and documented with one thing left to do: FIND the CURE!!!

So, our real journey has just begun!!! In September 2012, our family went to Finland to talk to the AGU doctors and families. Most of the work on AGU in the past 30 years were done in Finland. There we met with a family from France, whose daughter Hermance was diagnosed with AGU just a few months ago too. It really feels good to know that you are not alone in this world! During our trip we learned that most of the research efforts have stopped due to lack of funding and interest and no work is currently conducted to develop a cure for AGU. 

Equipped with the knowledge of the problem and no other place to go, we decided to work on a solution and save kids’ lives! We interviewed a lot of researchers and discussed the ideas about the development of AGU treatment. In December of 2012, we started a project with UNC to develop a treatment for AGU and to help our kids and other patients around the world. Currently, the treatment is in the pre-clinical studies.

Soon after we stated a non-profit 501(c)(3) organization with a mission to raise funds to offer a hope and eventual treatment to every person affected by AGU. We are developing the cure together with the fundraisers just like you.