Help find a cure for Christopher and other kids with ataxia-telangiectasia and #bethehope!

In 2017 our son Christopher was diagnosed with Ataxia, Telangiectasia, or A-T, a rare fatal genetic disease. A-T combines the worst symptoms of muscular dystrophy, cerebral palsy, cystic fibrosis, immune deficiencies, and cancer. A-T attacks in early childhood and is degenerative, affecting the immune system, nervous system, causing progressive loss of muscle control and lung disease. It also predisposes children to lethal cancers, like leukemia and lymphoma. Children with A-T are usually confined to a wheelchair by age 10 and often do not survive their teen years.

So far, no cure for A-T has been found and affected children around the world continue to face uncertain future. Recently, a new reason for optimism emerged. By applying a gene therapy approach called "antisense oligonucleotide", or 'ASO" therapy, researchers may now have an opportunity to produce the functional ATM protein that is missing within the cells of children with A-T.  The non-profit A-T Children's Project is behind the the first 'ASO" therapy trial currently taking place. If successful it will give hope to more children battling A-T. Initially, only the youngest kids who have lost fewer brain cells and who carry a certain type of mutation can be treated with this approach and a customized drug will be needed for each child. Despite these limitations, rescuing first few children from this terrible disease will be a huge leap from symptomatic treatment to potentially giving more children born with A-T a full, normal, healthy lifetime. 

A-T Children's Project partners with academic and industry investigators worldwide organizing and supporting innovative research, conferences, clinical teams, data platforms and biomarkers to optimize disease management strategies, develop new treatments and find a cure. By joining us and raising funds, you can be the hope for every child battling A-T!