Makeda is the only child in Canada with this ultra-rare, fatal disease. Your donation will give her, and future AGU kids, a fighting chance.
We are a non-profit organization with a mission to develop a cure for Aspartylglucosaminuria (AGU), a fatal genetic neuro-degenerative disease. We need to raise US$2 million for an upcoming clinical trial to treat this disease and we need your help to raise $500,000 of that here in Canada!
On the News
See this thrilling example of how gene therapy changes lives:
How a new gene therapy changed the lives of two Canadian girls
Campaign to save a child’s life
AGU is a rare genetic autosomal recessive disease that results in an inability of the body to breakdown fats, proteins and sugars. This process is necessary for appropriate mental and physical development. Initial symptoms are language/speech delay and clumsiness, which appear between two and five years of age. As the disorder progresses, language skills are affected the most. Prior to death, AGU causes intellectual disability which progressively worsens in adolescence. Most people with this disorder lose much of the speech they have learned and affected adults usually have only a few words in their vocabulary. Adults with AGU may develop seizures or problems with movement.
School-age children most often are diagnosed with an autism spectrum disorder and ADHD. Children continue to develop until their early twenties, but the learning occurs at a slower rate. An AGU child’s life expectancy usually averages between 25 and 35 years of age, with young adults typically dying from infection. There is currently no approved treatment for AGU.
Become a Volunteer
How can you help? Help us spread awareness so we can cure AGU kids and let families know they are not alone. We want to help as many people as possible and we can never have enough enthusiastic volunteers! Please contact us for more info.