A scientific article co-authored by Barbara Pietrucha MD, PhD, professor of the CMHI, has been published in the prestigious journal – Lancet Neurology.

Authors: Zielen Stefan, Crawford Thomas, Benatti Luca, Magnani Mauro, Kieslich Matthias, Ryan Monique, Meyts Isabelle, Gulati Sheffali, Borgohain Rupam, Yadav Ravi, Pal Pramod, Hegde Anaita, Kumar Suresh, Venkateswar Anand, Udani Vrajesh, Vinayan Kollencheri P., Nissenkorn Andreea, Fazzi Elisa, Leuzzi Vincenco, Stray-Pedersen Asbjorg, Pietrucha Barbara, Pascual Samuel I., Gouider Riadh, Koenig Mary Kay, Wu Steve, Perlman Susan, Thye Dirk, Janhofer Guenter, Horn Biljana, Whitehouse William, Lederman Howard
Title: Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial.
Journal: Lancet Neurology
2024 : Vol. 23, Nr 9, s. 871-882
IF: 46.500

The publication concerns an innovative method of treating patients with ataxia-telangiectasia (A-T) syndrome, involving the administration of dexamethasone in the patient’s own blood cells.

Currently, there are no effective treatments for A-T, which is why the discovery of a new therapeutic method is a huge breakthrough.

The new type of therapy not only alleviates neurological symptoms, as has been the case to date, but also slows down the progression of the disease by approximately 3.5 years, which significantly improves the functioning of the youngest patients. This discovery therefore brings about a real improvement in the quality of life of patients and their families.

Prof. Barbara Pietrucha has been involved in A-T research for many years, and her commitment goes far beyond scientific activity. In 2004, Prof. Pietrucha initiated the establishment of the „Razem Zdążymy” Foundation.

This publication is a significant contribution to the development of science and medicine.