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Article History

Received: 25 July 2022

Accepted: 19 December 2022

First Online: 9 January 2023

Declarations

:

: The study was approved by the ethics committees of all participating centres (Hospital Universitario Virgen del Rocío, Sevilla, Spain, Hospital Universitario Virgen de la Victoria, Málaga, Spain, Hospital Serranía de Ronda, Málaga, Spain, Fundación Puigvert, Barcelona, Spain, Hospital Clínic de Barcelona, Barcelona, Spain, Hospital Clínico Universitario de Santiago CHUS, A Coruña, Spain, Hospital Universitario Son Llàtzer, Illes Balears, Spain, Hospital Universitari Son Espases, Illes Balears, Spain, Hospital Clínico Universitario Virgen de la Arrixaca, Murcia, Spain, Hospital General Universitario de Alicante, Alicante, Spain, Hospital General Universitario de Elda, Alicante, Spain and Hospital General Universitario de Elche, Alicante, Spain). Written informed consent was obtained from all alive patients.

: Not applicable.

: RS has participated in advisory boards, clinical trials, and sponsored registries, and has received honoraria for lectures, attending meetings and/or travel, from Shire, Takeda, Amicus Therapeutics, Sanofi Genzyme, Eusa Pharma and Bayer. TRV has received honoraria for advisory roles from Amicus Therapeutics. MLM has received honoraria for lectures or educational events from Amicus Therapeutics, Sanofi Genzyme and Takeda-Shire, and has participated in advisory boards of Amicus Therapeutics, Sanofi Genzyme and Takeda-Shire. JJR has received honoraria for lectures and attending meetings and/or travel from Amicus Therapeutics, Takeda and Sanofi Genzyme, and for advisory roles from Amicus Therapeutics and Sanofi Genzyme. JRG has participated in advisory boards of Amicus Therapeutics and Sanofi Genzyme. AH is a study coordinator for clinical trials sponsored by Sanofi Genzyme and has received honoraria for presentations and advisory roles from Amicus Therapeutics, Sanofi Genzyme, Chiesi Global Rare Diseases and Takeda-Shire. AM has received honoraria for advisory roles from Alnylam, Amicus Therapeutics, Sanofi Genzyme, and Takeda-Shire. JMGP has received honoraria for advisory roles from Sanofi Genzyme and Takeda-Shire, and for lectures from Takeda-Shire, Amicus Therapeutics. EF has received honoraria for lectures from Takeda and attending meetings from Amicus Therapuetics. AAB is an employee of and holds stock in Amicus Therapeutics. RT has received honoraria for presentations and advisory roles from Amicus Therapeutics, Takeda, Sanofi and Chiesi. MARZ, and JVT have no competing interests to declare.