Document is current

Article History

Received: 29 May 2020

Accepted: 9 December 2020

First Online: 6 January 2021

Ethics approval and consent to participate

: All patients or caregivers provided informed consent to participate in the program.

: Consent was obtained to publish findings in an aggregated de-identified manner to protect patient privacy.

: AM has received consulting fees from Sanofi-Genzyme and Shire*, and research grants from Amicus, Protalix/Pfizer, Sanofi-Genzyme, and Shire*. UR has served on advisory boards for Amicus, Chiesi, Idorsia, Shire*, and Sanofi-Genzyme; has received honoraria from Amicus and Shire*; and received travel expenses from Amicus, Sanofi-Genzyme, and Shire*. JM has served on advisory boards for BioMarin, Denali Therapeutics, Green Cross, Regenxbio, Sanofi-Genzyme, and Shire*; and has received consulting fees from BioMarin, Denali Therapeutics, Eloxx, Green Cross, PTC Therapeutics, Regenxbio, Sangamo, Sanofi-Genzyme, and Shire*. RG has served on advisory boards for Amicus, BioMarin, GC Pharma, Inventiva, JCR Pharmaceuticals, Lysogene, Regenxbio, Sanofi-Genzyme, Shire*, Sobi, and Ultragenyx; has received research grants from Amicus, Armagen, BioMarin, GC Pharma, JCR Pharmaceuticals, Lysogene, Regenxbio, Sanofi-Genzyme, and Shire*; has received honoraria from Amicus; and has received travel expenses from BioMarin, JCR Pharmaceuticals, Sanofi-Genzyme, and Shire*. KU and TC-H have nothing to disclose. ZP and HW were employees of Shire International GmbH* at the time the study took place. JF has received consulting fees from Dicerna Pharmaceuticals and travel expenses from Shire*. *A Takeda company.