Declaration of interest
R Kaczmarek is serving on a scientific advisory board of BioMarin and received research funding from Bayer. RW Herzog is serving on the scientific advisory boards and committees of Regeneron Pharmaceuticals, Pfizer, BioMarin, Spark Therapeutics, Hoffman-La Roche, and Prevail Therapeutics.
The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Reviewer disclosures
A peer review on this manuscript has received prior research funding from Biomarin. prior advisory board for CSL Behring, Genentech, Sanofi Genzyme, and is currently an advisory board member for Regeneron.
A peer reviewer on this manuscript has received financial support for research from Anthos, Bayer, CSL Behring, Novo Nordisk, and Roche, as well as honoraria for lecturing or consultancy from Alexion, Bayer, CSL Behring, Daiichi Sankyo, Octapharma, Pfizer, Sobi, and Viatris.
Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.