As we head into 2022, research to find treatments for ataxia-telangiectasia (A-T) is moving more swiftly than ever. Please help us maintain momentum by making a year-end donation.
Researchers at Boston Children’s Hospital are now treating a little girl with A-T using antisense oligonucleotide (ASO) gene therapy and will soon be expanding their testing to more children. We are also funding other DNA-based approaches to overcome genetic mutations and restore a functional A-T gene in everyone with A-T.
In addition, we have built a next-generation data platform and harnessed cloud computing so that researchers can gain access to new data sets more quickly, enabling breakthrough discoveries. The cutting-edge data platform also shows global pharmaceutical companies as well as biotech startups that, while our kids’ disease is rare, our community of A-T families has assembled the tools and data that companies need to consider working on A-T. So far, A-T families from over 45 countries are sharing their data with researchers through this Global A-T Family Data Platform. And, besides contributing health data, children and young adults with A-T are providing blood samples and wearing sensors so that scientists can find biomarkers to objectively measure whether treatments are working.
With your help, researchers can make even greater strides in 2022. We won’t stop until we have a cure for all children and young adults with A-T. Please make a difference today by making a donation.