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Article History

Received: 28 June 2023

Accepted: 18 September 2023

First Online: 27 October 2023

Declarations

:

: All procedures followed were in accordance with the ethical standards of the responsible committee on human experimentation (institutional and national). This study was conducted in accordance with the Declaration of Helsinki of 1975, as revised in 2000, and Good Pharmacoepidemiology Practices (GPPs). The study protocols and questionnaire were approved by the Ethical Review Board of the local committee at all participating sites. Written informed consent was obtained from all individual participants included in the study. Eligibility of the participants were at the discretion of the physician and based on standard treatment options as determined by the Italian regulations (including AIFA authorization 341/2020 for Pompe disease patients and MPS I patients). Dosage and dosage regimen were in accordance with the Summary of Product Characteristics (SmPC).

: Not Applicable.

: VC, GC, RF, OM, RT, and AL declares no conflict of interest. RP received consulting fees from Sanofi. AS has received honoraria from Sanofi, Takeda, and Amicus Therapeutics. AF has received travel grants for participating in national and international meetings from Sanofi and Biomarin; and received honoraria for participating at advisory boards from Sanofi, Amicus and Takeda. M-SA has received honoraria and consulting fees from Sanofi, Takeda and Chiesi; also received grants from MetabERN, SIMMESN and SSIEM. EV has received honoraria and consulting fees from Sanofi. LM has received honoraria for participating at advisory boards or speaking at conferences and/or research support from Biogen, Roche, Sanofi, Alexion, Amicus, Janssen, and Lupin. M-SC has received honoraria, consulting fees, and/or research support for participating in conferences related to Pompe disease. SR received honoraria for participating at advisory boards from Sanofi and Amicus. SG received honoraria, travel grants and/or research support from Sanofi, Chiesi and Utragenyx. AT has received honoraria for participating at advisory boards or for educational talks from Sanofi, Amicus and Spark.