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Article History

Received: 31 May 2021

Accepted: 13 October 2021

First Online: 16 November 2021

Declarations

:

: Not applicable.

: Not applicable.

: G. Biasucci has received advisory board honoraria, speaker fees and travel support from BioMarin, SOBI, Vitaflo, PIAM Farmaceutici, Recordati Rare Diseases. A. Burlina has received advisory board honoraria, speaker fees and travel support from BioMarin, Nutricia, PIAM Farmaceutici, Sanofi Genzyme, Takeda Shire, Recordati Rare Diseases. M.T. Carbone has received advisory board honoraria and travel support from BioMarin, PIAM Farmaceutici, APR, Vitaflo. C. Cazzorla has received advisory board honoraria from BioMarin. V. Leuzzi has received advisory board honoraria, speaker fee and travel support from Nutricia, BioMarin, PTC, PIAM Farmaceutici. S. Paci has received advisory board honoraria, speaker fee and travel support from BioMarin, Nutricia, APR, Mamoxi, PIAM, Recordati Rare Diseases. F. Pochiero has received advisory board honoraria from BioMarin. M. Spada has received in the last two years advisory board honoraria, speaker fees and travel support from Sanofi, PIAM Farmaceutici, Takeda Shire, BioMarin, Nutricia, Sobi, Amicus, APR, Recordati Rare Diseases. A. Tummolo was invited advisor for BioMarin, Sanofi, Recordati and APR advisory boards. She received project funding from BioMarin. J. Zuvadelli has received advisory board honoraria, speaker fees and travel support from BioMarin, APR-Applied Pharma Research, Mamoxi and PIAM Farmaceutici.