Donate Now to Help Find a Cure for A-T!
WHAT IS A-T?
Imagine a disease that combines the worst symptoms of muscular dystrophy, cerebral palsy, cystic fibrosis, immune deficiencies, and cancer. Children with A-T are usually confined to wheelchairs by age 10 and often do not survive their teens. Because A-T is a multi-system disease, scientists believe that A-T research will help more prevalent diseases such as Alzheimer’s, Parkinson’s, AIDS, and cancer. With fewer than 500 reported cases of A-T in America, it is an orphan disease receiving very little medical attention, pharmaceutical interest, and research funding.
HOPE FOR A CURE
So far, no cure for A-T has been found and affected children around the world continue to face an uncertain future. Because of the efforts of the A-T Children's Project, a new reason for optimism emerged this year. By applying a gene therapy approach called "antisense oligonucleotide" or "ASO" therapy, reserchers may now have an opportunity to produce the functional ATM protein that is missing within the cells of children with A-T. Initially, only the youngest A-T kids who carry a certain type of mutation will be treated with this approach. But despite these limitations, rescuing the first few children from this terrible disease will be a huge leap from symptomatic treatment to potentially giving many more born with A-T a full, normal, healthy lifetime.
We hope you will join us in our race for a cure by making a donation to the A-T Children's Project!
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