Two laboratories have developed an innovation to fight Menkès disease, a hitherto incurable childhood pathology.
What if this was the solution to fight Menkes disease? The University of Lorraine (UL) has been seriously working on it since 2018. This disease is a rare pathology of genetic origin that causes a fatal copper deficiency in children, causing death before the age of 3.
Two laboratories at the University of Lorraine - the NGERE and the CITHEFOR - have been developing an alternative solution for four years now: a synthetic transporter capable of carrying copper to the brain. The first results obtained have been "very promising". "Biocompatible" and "non-toxic", this nanotransporter would be the answer to this disease for which there is currently no effective treatment.
An accelerated regulatory procedure is currently under discussion with the European Medicines Agency, with a marketing authorization application expected in 2023. A first French patent was filed in 2021 and its European extension is underway.