A-T Parents, While we are not aware of anyone with A-T in the world who has contracted COVID-19, the situation in our communities definitely impacts A-T families as they stay home to keep safe. To help families navigate this new normal, the A-T Children’s Project hosted a special webinar on Tuesday April 28th: COVID-19 and … Read More
The United Kingdom has come up with a “BRILLIANT” idea for something charities can do in the midst of this terrible COVID-19 environment to ensure that fund raising for important causes continues. And a wonderful organization based in England, Action for A-T, has encouraged our global community of A-T families, volunteers, donors and friends to … Read More
A-T Parents, Hope you and yours are keeping safe and well! With schools across the United States and Canada closed, A-T parents are stepping up to manage homeschooling their kids.To help parents navigate this new normal, the A-T Children’s Project hosted a special webinar on Saturday, April 18th. Over 50 families tuned in as A-T … Read More
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
Dear A-T Families, The A-T Children’s Project is eager for families of children and adults who have A-T to be as informed as possible as we all plan for the emerging COVID-19 virus outbreak. We strongly suggest that A-T families review the information that is provided by the Centers for Disease Control and Prevention. It … Read More
We are excited to announce that we are selling six new shirts for Rare Disease Day 2020, coming up on February 29th. We’ve chosen the Be Brave, Be Strong, Be Proud slogan this year as a testament to the fortitude of A-T families. The shirts are available starting today in both grey and white at … Read More
Thank you for your amazing generosity on #GivingTuesday this year! It’s astounding what can happen when we pull together to help find a cure for A-T. For #GivingTuesday, we started off slowly, but a surge of kindness just before the deadline enabled us to maximize the $50,000 matching gift, doubling the impact of your … Read More
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
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
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
