Research and Association Aims
Researchers studying this pathology proceed in various directions with the final aim of finding medicines to stop progression of this disease. However, this pathology is a somewhat rare and like other genetic disease that the pharmaceutical companies are not motivated to invest in. Hopeful project costs are very high: a medicine scanning robot costs 400 dollars per minute, a researcher costs about $ 50.000 dollars per year and testing new substances about $ 130.000 dollars. It is important to know that ATM gene identification in 1995 (the first step to understand molecular basis' disease) was largely realized through an USA A-T child's family who raised contributions of about 3 million dollars.
While the first and only concern of "A-True Smile" is to subsidize valid research that will find the cure for Ataxia-Telangiectasia. Finding that cure may also lead to breakthroughs in other neurodegenerative diseases, thereby reaching the broadest possible benefit to mankind in general.
Researches done till today permitted to make the pathology knowledge larger and to find specific inner working opening ways to new possible therapy to approach the disease.
An efficient therapy to arrest Ataxia progression does not yet exist. Trials with antioxidants are ongoing because genetic A-T fault suggests cellular sensitivity to "oxidative stress", including Alpha-lipolic acid. However it is palliatives.