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Article History

Received: 17 January 2025

Accepted: 16 April 2025

First Online: 8 May 2025

Declarations

:

: This study did not require research ethics committee approval because it involves secondary analysis of anonymized aggregate data that adheres to recognized anonymization standards and does not contain sensitive information (e.g., clinical or genetic data). The study complies with all applicable ethical guidelines and data protection regulations. Additionally, it was conducted in accordance with the Helsinki Declaration of 1964, its subsequent amendments, and equivalent ethical standards where applicable.

: Not applicable.

: MM has received honoraria for speaking, advisory boards and compensation for congress participation from Sanofi Genzyme, Khondrion, Biogen, UCB, Stealth and Precision Biosciences, outside the submitted work. LM has received honoraria for speaking, advisory boards and compensation for congress participation from Sanofi Genzyme, Roche, Biogen, Amicus Therapeutics, Alexion, Argenx, UCB, Janssen, Lupin, outside the submitted work. PV received honoraria, consultation fees, and compensation for advisory board participation from Pfizer, Sanofi Genzyme, CSL Behring, LFB, Natus, UCB Pharma, Janssen, Alnylam. VS received compensation for consulting services and/or speaking activities from AveXis, Cytokinetics, Italfarmaco, LiquidwebS.r.l., Novartis Pharma AG, Amylyx Pharmaceuticals, Biogen, and Zambon Biotech SA. MdV is a consultant to Novartis, Argenx and Astra Zeneca. CK acknowledges participation in Advisory Boards of Amicus Therapeutics, Chiesi, Fulcrum Therapeutics, Hormosan, Roche Pharma AG, Sanofi Genzyme, Stealth BioTherapeutics, and participation as local PI in clinical trials by Amicus Therapeutics, Fulcrum Therapeutics, Omeicos, Reneo Pharmaceuticals, Stealth Therapeutics, Scholar Rocks, and received travel grants and/or lecture fees from Amicus Therapeutics, Chiesi, Sanofi Genzyme, Argenx, Novartis, Santhera, Sarepta, UCB. DP acknowledges participation in Advisory Boards of Inflectis, Alnylam, Akcea, Arvinas, Augustine Tx, DTx, Novartis, participation as local PI in clinical trials sponsored by Alnylam, Ionis, AT Therapeutics, Nido-Biosciences. The remaining authors declare that they have no competing interests.