The start of the new year is less than 24 hours away, and for families impacted by A-T, 2019 promises to be a year unlike any other.
As you may know from our recent updates, we’re on track to deliver a gene therapy drug (an antisense oligonucleotide) to an 18-month-old girl with A-T who lives in northern California. If that drug works as intended, cells within this little girl’s brain will begin producing the ATM protein that’s been missing, and her future will be transformed. We’re not ready to use the word “cure,” but we’re now manufacturing a drug that, even two years ago, none of us dared dream possible.
As an “orphan disease” charity, most of the research we support has been funded not by large corporate donations or grants but many smaller donations from friends and family. Because of scientific discovery projects as well as clinical studies funded by those past donations, improved disease management strategies have extended the average life span of A-T children from their mid-teens to their mid-twenties, enabling literally hundreds if not thousands of extra birthdays that would never have occurred. And now, we’re about to take advantage of new technologies, starting with ASO therapy for some A-T children but hopefully followed soon by additional approaches that will make it possible to help every child and young adult with A-T.
We thank you for everything you have previously done for us. And, if your year-end gift planning has left room for any remaining donations, we hope you’ll keep us in mind now. Every dollar matters, and bold progress is happening!
Our best wishes to you and your family for a safe, healthy and prosperous new year.