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 $3.5 million to pay for the medicine that will be used in a clinical trial next year in the United States.
We will need an additional funding to pay for their hospital bills. It will mount to another 1.5-2 MM$.
Aspartylglucosaminuria (AGU)
Diseases often occur due to a faulty or missing gene. One way to cure these diseases is to go after that gene, particularly trying to replace or alter it.
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.
Aspartylglucosaminuria or AGU for short, is an ultra-rare genetic disease belonging to a group of lysosomal storage disorders. Lysosomes are cellular compartments containing enzymes, responsible for the final (cellular) breakdown of fats, proteins and sugars.
A malfunction of this enzyme leads to the physical and neurological disease manifestations.