AUMC and VU have worked together with industry to develop animal and human disease models for STXBP1-RD and to design and test new therapies. In parallel, ESCO aims to prepare the field for successful and efficient evaluation of new therapies for STXBP1-RD and promote equal and evidence-based access to new therapies. Starting Q1 2024, ESCO will direct a natural history study (NHS) in eight EU sites + Israel. ESCO has secured its first funding from the international STXBP1 foundation. Several letters of intent have been signed by pioneer sponsors (industry). In parallel, ESCO has applied for the Innovative Tools program at EMA to be able to exploit the NHS as a concurrent control arm for future clinical trials.
ESCO was established in 2021 by Matthijs Verhage (VU/AUMC) and Ganna Balagura (Gaslini Hospital Genua, IT). Together with Andrea Soto Padilla, business developer at IAO/IXA Neuro, they form ESCO’s executive board. The eight partner countries are NL, BE, IT, DK, DE, FR, SP and IL. See www.stxbp1eu.org for more information.
