Updates are available Correction dated 2024-08-30

Article History

Received: 18 December 2023

Accepted: 1 July 2024

First Online: 9 July 2024

Change Date: 30 August 2024

Change Type: Correction

Change Details: A Correction to this paper has been published:

Change Details: https://doi.org/10.1186/s13023-024-03305-8

Declarations

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: This study was conducted in accordance with the Declaration of Helsinki of 1975, as revised in 2000, conducted in full conformance with the principles of the ‘Declaration of Helsinki’ (64th WMA General Assembly, Brazil, October 2013), Good Clinical Practice guidelines as appropriate to nutritional products, and with local legislation of the country in which the research is conducted, whichever affords the greater protection to the individual. The study protocols and questionnaire were approved by the Ethical Review Board of the local committee at all participating sites. Written informed consent was obtained from all individual participants included in the study. Eligibility of the participants were at the discretion of the physician. In the United Kingdom (UK) ethical approval was received from the NRES Committee London- Queen Square. Ref: 15/LO/0685, IRAS project ID 148762. In France a favourable opinion was received from Comite de Protection des Personnes (CPP) Ile de France XI, CPP ref: 16012. Ethical approval was received from the Institutional Review Board (IRB) of Connecticut Children’s Medical Centre, CCMC IRB# 16–101. In the Netherlands a favourable opinion was received from Universitair Medisch Ethische Toetsingscommissie: University Medical Centre Groningen (METc UMCG). It opened in six international sites and was registered at (NCT02318966).

: All participants provided written informed consent before participating in the study. The authors have all approved publication in the <i>Orphanet Journal of Rare Diseases</i> and agree to transfer of rights to the publication.

: KMMCK and EAB are employees of Vitaflo International Ltd. All the investigators and sites received grant support for these studies from Vitaflo International, Ltd. DAW also serves as a consultant for Weinstein Rare Disease and Clinical Development Consulting, LLC. JED, MW, PL, EM, KMR, CV, & US have no competing interests. AJMW, JG & CE have received honoraria, research funding and travel expenses from Vitaflo International Limited and Nutricia. HRM has received honoraria, research funding and travel expenses from Vitaflo International Limited and Nutricia. A contract is in place as an advisor with Ultragenyx Pharmaceutical Inc and Kriya Therapeutics for gene therapy studies in GSD. TGJD declares that he has confidentiality agreements with third parties. In the past 36 months, there have been consultation agreements (with Danone, Ultragenyx Pharmaceutical Inc, ModernaTX Inc, and Beam Therapeutics), contracts for financial research support for investigator-initiated research (NCT04311307) and sponsor-initiated research (NCT03517085, NCT03970278, NCT05139316, and NCT05196165), honoraria for lectures or presentations (by MEDTalks, Prelum, and Danone), and participations in a Data Safety Monitoring Board (NCT05095727) and Advisory Boards (Ultragenyx Pharmaceutical Inc, ModernaTX Inc, and Beam Therapeutics). For all private-public collaborations, including this sponsor-initiated clinical trial, contracts are via UMCG Contract Research Desk, and all payments are to UMCG. FdB declares she has confidentiality agreements with third parties. There have been contracts for financial research support for investigator-initiated research (NCT04311307) and sponsor-initiated research (NCT03517085, NCT03970278, NCT05139316). For all private-public collaborations, including this sponsor-initiated clinical trial, contracts are via UMCG Contract Research Desk, and all payments are to UMCG.