EPTRI participated to the 6th edition of the RE(ACT) Congress and the 4th edition of the IRDiRC Conference that took place online from the 13th to the 15th of January 2021. It was organised by BLACKSWAN Foundation in collaboration with the European Joint Program on Rare Diseases (EJP RD) and in partnership with EURORDIS (European alliance of patient organizations) and RDI – Rare Diseases International (global alliance of people living with a rare disease).
It represented the occasion to share cutting- edge research on rare and orphan diseases with the ultimate goal of enhancing the rapid delivery of new and promising diagnostics and therapies to patients all around the world, promoting exchange of ideas and discuss policies related to rare diseases research.
They approached several themes related rare diseases in terms of application of diagnostic, artificial intelligence, new technologies and advanced therapies.
In that occasion, EPTRI submitted an abstract “Paediatric oral liquid formulations of sodium dichloroacetate for the acute and chronic treatment of acquired or congenital lactic acidosis” in the category “Access to diagnostic and drugs for all” which has been included in the Abstract Book of the events. The abstract is focused on the application of the sodium dichloroacetate (DCA) in acute and chronic treatment of acquired and congenital lactic acidosis in the paediatrics, specifically to identify stable palatable solutions containing excipients tolerated by paediatric patients and orally disintegrating mini-tablets of sodium DCA as an alternative suitable for children formulation and therefore represent a relevant pilot case to reduce off-label use in children.
If you want to read our abstract, you can find it in the Abstract Book at this link (page 84).