Avalglucosidase alfa in infantile-onset Pompe disease: A snapshot of real-world experience in Italy

Autor: Fiumara, Agata, Sapuppo, Annamaria, Gasperini, Serena, Crescitelli, Viola, Sacchini, Michele, Procopio, Elena, Gragnaniello, Vincenza, Burlina, Alberto
Zdroj: In Molecular Genetics and Metabolism Reports September 2024 40
Databáze: ScienceDirect