Hannah, William B. http://orcid.org/0000-0003-1643-0737
Derks, Terry G. J.
Drumm, Mitchell L.
Grünert, Sarah C.
Kishnani, Priya S. http://orcid.org/0000-0001-8251-909X
Vissing, John
Article History
Accepted: 31 July 2023
First Online: 7 September 2023
Competing interests
: W.B.H. has received consulting fees from PTC Therapeutics and ReCode Therapeutics. T.G.J.D. declares that he experiences no competing interests concerning the content of this manuscript. However, there are 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 participation in a Data Safety Monitoring Board (NCT05095727) and advisory boards (Ultragenyx Pharmaceutical, Inc., ModernaTX, Inc. and Beam Therapeutics). For all private–public relationships, all contracts are via UMCG Contract Research Desk and all payments are to UMCG. S.C.G.: the Centre for Paediatrics and Adolescent Medicine Freiburg received funding for the following sponsor-initiated study, for which S.C.G. is a local subinvestigator: NCT05139316 – ‘a study of adeno-associated virus serotype 8-mediated gene transfer of glucose-6-phosphatase in patients with glycogen storage disease type Ia (GSDIa)’, sponsored by Ultragenyx Pharmaceutical, Inc. S.C.G. has received honoraria for educational lectures from Vitaflo GmbH and Ultragenyx Pharmaceutical Inc. and support for attending metabolic expert meetings from Nutricia Metabolics GmbH. S.C.G. participated in an advisory board for Ultragenyx Pharmaceutical, Inc. P.S.K. has received research/grant support from Sanofi Genzyme, Amicus Therapeutics and Kriya Therapeutics. P.S.K. has received consulting fees and honoraria from Sanofi Genzyme, Amicus Therapeutics, Maze Therapeutics, JCR Pharmaceutical, Asklepios Biopharmaceutical, Inc. (AskBio), Ultragenyx Pharmaceutical, Moderna, Inc. and Kriya Therapeutics. P.S.K. is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Amicus Therapeutics and Baebies. P.S.K. has equity in Asklepios Biopharmaceutical, Inc. (AskBio), Maze Therapeutics, and equity options with Kriya Therapeutics. J.V. has received honoraria for acting as speaker or consulting for Sanofi Genzyme and Amicus Therapeutics and has participated in a clinical trial sponsored by Sanofi Genzyme without getting honorarium. M.L.D. has no competing interests to declare relevant to this article.