Functional assessment tools in children with Pompe disease: A pilot comparative study to identify suitable outcome measures for the standard of care

Federica Ricci, Chiara Brusa, Francesca Rossi, Enrica Rolle, Valeria Placentino, Angela Berardinelli, Veronica Pagliardini, Francesco Porta, Marco Spada, Tiziana Mongini

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Medicine & Life Sciences