More than 40 participants from Academia, Industry and Representatives of patients and parents attended the first Work Package 4 (Scientific Advice, Feasibility and Innovation) face to face meeting. The event was co-organised by Radboud University Medical Center Nijmegen (RUMC) and Fondazione PENTA, on 18th September 2018 at Schiphol Airport, Amsterdam. Chaired by Saskia N. de Wilt of RUMC, the current status of Work Package 4 was discussed at length. Among the presentations included were examples of how clinical expert and methodology expert groups can be set up as well as patients’ and parents’ involvement. It was the perfect occasion to define efficient synergies and for partners to reaffirm their full commitment to the planned work ahead, representing a key opportunity to pave the way for the success of Work Package 4 moving forward.

We extend our thanks to all participants involved!