Research Updates
Hi everyone, I hope you and your family are doing well and keeping safe. As much as the COVID-19 pandemic has changed our world, I wanted to let you know that our ASO gene therapy trial for A-T – that you helped make possible – has not stopped. It continues…
MoreMichel Willemsen, MD, PhD and his colleagues at Radboud University in the Netherlands have begun an “open-label” clinical trial of nicotinamide riboside in children and young adults with ataxia telangiectasia (A-T). With financial support from the A-T Children’s Project, Dr. Willemsen’s team is currently enrolling 32 individuals with A-T in…
MoreAs this historic milestone approaches, we can’t help but feel a renewed sense of urgency to reach our goal. And, we never forget for even a moment that this project would not be happening without the wonderful support we’ve received from you. More soon, Brad Margus Founder
MoreHi Everyone, It’s settled. We’ve selected a little, 18-month girl on the west coast of the U.S. to be the first A-T child in history to receive gene therapy. We had the pleasure of letting her parents know the exciting news last week. How did we pick the first kid?…
MoreLiver problems, such as non-alcohol fatty liver, fibrotic changes and even non-alcoholic cirrhosis, have been seen as children with A-T become adults. In addition, liver disease has been associated with insulin resistance, which is also seen in A-T patients. To better understand liver disease in A-T by investigating the correlation…
MoreEmail from Volunteer Chairman and Founder, Brad Margus: As you know, to develop an urgently needed treatment for A-T, we’ve been eager to use a gene therapy approach called “antisense oligonucleotide” or “ASO” therapy to produce a functional ATM protein in children who have A-T. While (with your help!) we’ve…
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