Yap, Sufin
Lamireau, Delphine
Feillet, Francois
Ruiz Gomez, Angeles
Davison, James
Tangeraas, Trine
Giordano, Vincenzo
Clinical trials referenced in this document:
Documents that mention this clinical trial
Real-World Experience of Carglumic Acid for Methylmalonic and Propionic Acidurias: An Interim Analysis of the Multicentre Observational PROTECT Study
https://doi.org/10.1007/s40268-023-00449-z
Funding for this research was provided by:
Recordati Rare Diseases
Article History
Accepted: 30 October 2023
First Online: 10 January 2024
Declarations
:
: This study, medical writing assistance and the open access fee were funded by Recordati Rare Diseases. The funding source was involved in the writing of the manuscript.
: Sufin Yap has received funding for conferences and honorarium for lectures, master classes, advisory boards and ‘meet the expert’ sessions from Recordati Rare Diseases; and honorarium for expert advice from OpenVie. Vincenzo Giordano is an employee of Recordati Rare Diseases. Delphine Lamireau, Francois Feillet, Angeles Ruiz Gomez, James Davison, and Trine Tangeraas have no conflicts of interest that are directly relevant to the content of this article.
: The study is being conducted in accordance with the ethical standards of the institutional and/or national research committees at each centre and with the 1964 Declaration of Helsinki, as revised in 2013. The study protocol has been approved by the local institutional review board at each study site.
: Written informed consent is being collected from all participants or their caregivers before initiating the study.
: Not applicable.
: The datasets used and/or analysed during the current study are available from the corresponding author upon reasonable request.
: Not applicable.
: SY, DL, FF, ARG, JD and TT enrolled patients and contributed to the interpretation of the results, and VG contributed to the study design and interpretation of the results. All authors reviewed and revised the manuscript for important intellectual content, approved the version to be published, and agree to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.