Research Updates

Making Strides in A-T Research – Thanks to You

Dec 31, 2020 Comments Off on Making Strides in A-T Research – Thanks to You
Jun 21 @ 1:02 am

Dear Friends, This past year has been difficult for so many people. Every A-T family I have spoken with has been enormously grateful that, thanks to many of you, research has continued, providing the hope that sustains us. Here’s a quick look at our efforts this year: With the start…

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A Biomarker to Track Brain Cell Death in A-T Kids

Jun 4, 2020 Comments Off on A Biomarker to Track Brain Cell Death in A-T Kids
Jun 21 @ 1:02 am

Hi everyone, I hope you and your families are continuing to stay safe during the pandemic. Over the past year, you’ve heard me talk a lot about testing an approach called antisense oligonucleotide (ASO) gene therapy in a little girl with   A-T. She has continued to receive regular injections, and…

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A-T ASO Gene Therapy Trial Continues!

Apr 17, 2020 Comments Off on A-T ASO Gene Therapy Trial Continues!
Jun 21 @ 1:02 am

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…

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A Clinical Trial of Nicotinamide Riboside (Niagen®) in A-T

Jun 25, 2019 Comments Off on A Clinical Trial of Nicotinamide Riboside (Niagen®) in A-T
Jun 21 @ 1:02 am

Michel 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…

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FDA Approves Clinical Testing of an Amino Acid in A-T

Apr 4, 2019 Comments Off on FDA Approves Clinical Testing of an Amino Acid in A-T
Jun 21 @ 1:02 am
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Update on Testing ASO Gene Therapy in A-T Kids

Mar 25, 2019 Comments Off on Update on Testing ASO Gene Therapy in A-T Kids
Jun 21 @ 1:02 am

As 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

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How Do Cells Change and Die in the Human A-T Brain?

Mar 18, 2019 Comments Off on How Do Cells Change and Die in the Human A-T Brain?
Jun 21 @ 1:02 am
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A-T Child and Oligo Selected for ASO Gene Therapy

Nov 30, 2018 Comments Off on A-T Child and Oligo Selected for ASO Gene Therapy
Jun 21 @ 1:02 am

Hi 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?…

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Liver Disease in A-T

Nov 20, 2018 Comments Off on Liver Disease in A-T
Jun 21 @ 1:02 am

Liver 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…

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Non-Viral Gene Therapy for A-T

Sep 15, 2018 Comments Off on Non-Viral Gene Therapy for A-T
Jun 21 @ 1:02 am
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