(ANSA) – URBINO, 21 AUG – A breakthrough in the treatment of ataxia telangiectasia, a rare hereditary disease that affects children and involves many organs and body systems, thanks to the Carlo Bo University of Urbino.
The results of the study that explored the innovative use of red blood cells in the treatment of the pathology in question, the largest global clinical study that uses the technology developed by the University of Marche, were published last August 15 by Lancet Neurology, the most important journal in the world in the field of neurology and in particular clinical neurology.
Conducted in 22 centers distributed in 12 countries on 5 continents, the study involved 175 patients aged 6 years and older. The technology used in this clinical study was discovered and patented by the group active at the University of Urbino led by Professor Mauro Magnani who, together with Professor Luigia Rossi, founded the start up EryDel Spa in 2007.
“The patients – explains Professor Magnani – were divided to receive the treatment or a placebo, showing a significant efficacy in children aged between 6 and 9 years, without the side effects usually associated with corticosteroids”.
EryDel has brought this innovative therapeutic approach to the clinic with the support of venture capital funds and some public funding. The asset was recently acquired by Quince Therapeutics (listed on the stock exchange as Nasdaq: Qncx), which has started a further study and received from the Food and Drug Administration, the agency responsible for evaluating and regulating drugs in the United States, the recognition of a fast track designation, that is, a path useful to facilitate the development and accelerate the review process of treatments under experimentation.
Scientific and economic efforts are all aimed at making this technology accessible to all patients, who will finally have access to a specific treatment. “These results – concludes Magnani – represent a milestone in the treatment of ataxia telangiectasia.
We have demonstrated that it is possible to improve the quality of life of our youngest patients with an effective and safe therapy”. (ANSA).
2024-08-22 06:16:18