Adrestia Therapeutics Partners with Patient Advocacy Groups to Further A-T Research
The A-T Children's Project is excited to be part of this A-T research initiative. Please see the following press release from Adrestia Therapeutics: Read More
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
Promising Study Finds Ways to Measure A-T
Dear Friends, We are excited to share the following update from Dr. Anoopum Gupta to the A-T families who have been participating in his project to find better ways to measure A-T. We are so grateful to him, to the families, and, of course, to our donors and fundraisers who help to make it all … 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-T ASO Gene Therapy Trial Continues!
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 forward, bringing hope to many … 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
Update on Testing ASO Gene Therapy in A-T Kids
It’s been several months since I last updated you on our progress toward testing an antisense oligonucleotide (ASO) gene therapy in a child with A-T. In this update, I thought I’d go a little deeper on a couple aspects of our development efforts, including some challenges we’ve recently overcome and some opportunities that have emerged … Read More