“Innovative approaches to develop paediatric drugs for iNMDs”, the first InNerMeD e-learning course

InNerMeD is glad to announce the launch of the first of a series of e-learning courses that will be produced in the framework of the InNerMeD project.

The first course has been held by Dr. Giovanni Smania from Consorzio per Valutazioni Biologiche e Farmacologiche, Bioinformatics, Mathematical Modelling and Synthetic Biology Lab, University of Pavia and Dr. Viviana Giannuzzi from Gianni Benzi Pharmacological Research Foundation and is entitled Innovative approaches to develop paediatric drugs for iNMDs. The lecture is focused on the application of innovative methodologies that can facilitate the approval of new drugs for the treatment of inherited neurometabolic disorders in the vulnerable population, since the onset of this class of diseases is usually during childhood, which makes clinical trials even more problematic to perform.

The innovative methodologies analysed in this lecture encompass pharmacokinetic-pharmacodynamic (PK-PD) modelling and simulation, biomarkers and innovative study designs in the context of an extrapolation approach, starting from the ICH E 11 guideline that provides recommendations on what type of studies should be performed in children based on the differences/similarities with the adult population.

All the e-learning courses will be available at the dedicated InNerMeD YouTube channel and published in the “E-learning” section of the InNerMeD project website.

Stay tuned with our website to attend the forthcoming e-learning courses that will be soon available free of charge:

  • “Rare Diseases”
  • “LSD differential diagnosis”
  • “iNMDs signs and symptoms”.