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doi: 10.1016/j.biopha.2020.110625 568 GhelardiEMazzantiniDCelandroniFCalvigioniMPanattoniALupettiAet al

Additionally, recent studies from 2024 to 2025 on NGPs highlight the urgent requirement for clearer guidance and harmonized approval procedures (156, 539)

However, a majority of the patients reach adulthood without any other symptoms than decreased stamina and fatigue 20,21