van Kuilenburg, André B. P. http://orcid.org/0000-0002-7989-4910
Hollak, Carla E. M.
Travella, Ana
Jacobs, Melisa
Gentilini, Lucas D.
Leen, René
der Vlugt, Karen M. M. Ghauharali-van
Stet, Femke S. Beers
Goorden, Susan M. I.
van der Veen, Sanne
Criscuolo, Marcelo
Papouchado, Mariana
Funding for this research was provided by:
Biosidus
Vriendenloterij
Article History
Accepted: 29 March 2023
First Online: 21 April 2023
Declarations
:
: This work is funded by Biosidus in collaboration with Pharmagister and the platform Medicine for Society for which funding is provided by the Vriendenloterij. None of the funders had any influence on the analysis and interpretation of the data.
: Ana Travella, Melisa Jacobs, Lucas D. Gentillini, Marcelo Criscuolo, and Mariana Papouchado are employed by Biosidus, which manufactures AGABIO. The remaining authors declare no conflict of interest.
: The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.
: Written informed consent was obtained to use patients fibroblasts for the study as part of our biobanking protocol, approved by the Ethics Committee.
: Not applicable.
: Not applicable.
: Not applicable.
: André B. P. van Kuilenburg, Carla E. M. Hollak, Marcelo Criscuolo and Mariana Papouchado designed the study. Ana Travella, Melisa Jacobs, Lucas D. Gentillini, René Leen, Karen M. M. Ghauharali-van der Vlugt, Femke S. Beers Stet, Sanne van der Veen, and Mariana Papouchado gathered the data. André B. P. van Kuilenburg, René Leen, Karen M. M. Ghauharali-van der Vlugt, Femke S. Beers Stet, Susan. M. I. Goorden, Sanne van der Veen, and Mariana Papouchado analyzed the data. André B. P. van Kuilenburg, Carla E. M. Hollak, René Leen, Karen M. M. Ghauharali-van der Vlugt, Susan. M. I. Goorden, Sanne van der Veen, Marcelo Criscuolo, and Mariana Papouchado wrote the publication. All authors read and approved the final version