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Article History

Received: 1 December 2023

Accepted: 30 March 2024

First Online: 13 April 2024

Declarations

:

: This study was conducted in accordance with the Declaration of Helsinki and all subsequent amendments, the guidelines for Good Epidemiological Practice, and Good Pharmacoepidemiology Practices issued by the International Society for Pharmacoepidemiology. All study documents were reviewed by the leading Ethics Committee (Landesärztekammer Hessen) and the Local Ethics Committee (Ärztekammer Hamburg). The committees had no concerns about the conduct of the study. No additional local review was needed for sites located in Berlin. All patients and/or patients’ parents/legal guardians provided informed consent or assent to participate in the study. For data collection from deceased patients, an informed consent waiver was used.

: EM has received research grants, speakers’ honoraria, and consultation fees from Sanofi, Takeda, Orphazyme, Avrobio, Idorsia, Cyclo Therapeutics, Alexion, and Amicus. NM has received research grants, speakers’ honoraria, and/or consultation fees from Sanofi, Takeda, Amicus, Chiesi, JCR, and Lysogene. NW has received research grants, speakers’ honoraria, travel grants, and/or consultation fees from Sanofi, Takeda, and Biomarin. AZ has received speakers’ honoraria and consultation fees from Sanofi, Genzyme, Takeda, and Biomarin. JN has received travel grants from Sanofi, Takeda, and Biomarin. BA and LL are employees of IQVIA. MG, SG, MF, and RPJ are employees and may hold stock and/or stock options in Sanofi. FL was an employee of Sanofi at the time of study conduct.

: Not applicable.