Ataxia-Telangiectasia
“ay-TACK-see-uh tel-LAN-jick-TAY-sha”

A-T may be very rare, but an incredibly dedicated group of friends and families are working together to accelerate research in our efforts to find life-improving therapies and a cure.

Help us find a cure for A-T by making a one-time donation or a monthly gift.

Unlock $100 for the A-T Children’s Project when you start a monthly gift of $10 (or more). The Wasie Foundation will donate $100 for each of the first 50 new monthly gifts in 2021.

 

While this has been an extremely difficult year for everyone, we are forging ahead thanks to the incredible dedication of our families, fundraisers, researchers, and donors.

We recently told you about our treating a little girl who has A-T with an antisense oligonucleotide (ASO) gene therapy drug, and also that we are finding a way to measure neurodegeneration in A-T with a simple blood test. Please know that we are working hard in other areas, too. During the past few weeks, we’ve been recruiting people with A-T for exciting new clinical studies. One is to test a compound called N-Acetyl-L-Leucine in a clinical trial for kids with A-T ages six and up which may improve how kids with A-T move. The second is a study to find a way to objectively measure how people with A-T move by using sensors in a wearable device that looks like a watch.

We promise to keep you posted as we continue to make progress, and thank you for helping to make it happen!

All the best,

Jennifer Thornton, Executive Director

 

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Ataxia-telangiectasia (A-T) is a genetic disease that causes loss of muscle control and balance, cancer, lung disease and immune system problems in children and young adults, shortening their lives. The nonprofit 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.

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