van Spronsen, Francjan J.
Blau, Nenad
Harding, Cary
Burlina, Alberto
Longo, Nicola
Bosch, Annet M.
Article History
Accepted: 30 March 2021
First Online: 20 May 2021
Competing interests
: F.J.v.S. has been a member of scientific advisory boards for defects in amino acid metabolism of APR, Agios, Arla Food International, BioMarin, Eurocept Int, Lucana, Moderna TX, Nutricia, Rivium, Homoly and Nestlé-Codexis; his institute has received research grants from Alexion, Biomarin, Codexis, Nutricia, SoBi and Vitaflo; his institute has received grants from patient organizations ESPKU, Metakids, NPKUA, Stofwisselkracht, Stichting PKU research and the Tyrosinemia Foundation; and his institute has received honoraria as consultant and speaker from APR, Pluvia, Biomarin, MendeliKABS and Nutricia. N.B. has received honoraria and/or consulting fees from BioMarin, Pharmaceuticals, Censa, Nestlé Pharmaceuticals and Homology Medicines. C.H. has received consulting fees, speaker fees, and travel and research support from BioMarin, Cydan Development Inc., Dimension Therapeutics, Horizon Pharma, Pfizer, Rubius Therapeutics, StrideBio and Synlogic. A.B. has received advisory board honoraria, speaker fees and travel support from Biomarin Pharmaceuticals, Nutricia, Cambrooke, PIAM, APR, Sanofi Genzyme and Takeda. N.L. has received consulting fees from Aeglea, BioMarin, Censa Pharmaceuticals, Dimension Therapeutics, Genzyme/Sanofi, Hemoshear, Horizon, Lumos Pharma, Moderna, Mitobridge, Pfizer, Retrophin and Stealth Therapeutics, and has conducted contracted research for Aeglea, BioMarin, Genzyme/Sanofi, Horizon, Lumos Pharma, Protalix, Retrophin, Shire, Stealth Therapeutics and Ultragenyx. A.M.B. has received a speaker fee from Nutricia and has been a member of advisory boards for Biomarin.