Giving Tuesday 2022

Dear Friends of the A-T Children’s Project, Giving Tuesday is tomorrow, and an anonymous donor has offered to match the first $50,000 in donations we receive! Please consider donating as we need your support more than ever. Why especially now? So that we can seize opportunities that have emerged from the remarkable progress we’ve seen … Read More

Drug Repurposing Screen for A-T Reaches Important Milestone

Charles River Lab, a drug development services company collaborating with the A-T Children’s Project, has succeeded in developing a laboratory test or “assay” to rapidly evaluate thousands of approved drugs to learn if some of them can be repurposed for A-T. Because drugs emerging from the screening process with promising results will have already been … Read More

Making Strides in A-T Research – Thanks to You

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 of our antisense oligonucleotide (ASO) … Read More

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

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 we’re grateful that we haven’t … Read More

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

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 the Netherlands in a four-month … Read More

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

IntraBio, a UK-based biopharmaceutical company, has received approval from the US Food and Drug Administration to run a clinical trial in patients who have Ataxia-Telangiectasia (A-T). The company will test their investigational new drug, IB1001, an orally-administered version of N-Acetyl-L-Leucine, to evaluate its safety and efficacy in improving symptoms, functioning, and quality of life. N-Acetyl-L-Leucine … Read More