Funding for this research was provided by:
Received: 1 February 2018
Accepted: 27 June 2018
First Online: 4 July 2018
Ethics approval and consent to participate
: All individuals consented to participate in the study, which was conducted in accordance with the Declaration of Helsinki of 1975, as revised in 2008.
: Not applicable.
: JV, BKB, RZ, JAT, MPW, and NL are investigators of BioMarin Pharmaceutical Inc. clinical trials, and RZ, JAT, and NL are on the steering committee for the pegvaliase clinical program. NL reports grants (Aeglea, BioMarin, Genzyme, Horizon, Lumos Pharma, Protalix, Retrophin, Shire, Stealth Therapeutics, Ultragenix), consultant fees (Aeglea, BioMarin, Censa Pharmaceuticals, Dimension Therapeutics, Genzyme, Hemoshear, Horizon, Lumos Pharma, Moderna, Mitobridge, Pfizer, Retrophin, Stealth Therapeutics), participation in clinical trials (Aeglea, BioMarin, Genzyme, Horizon, Protalix, Retrophin, Shire, Stealth Therapeutics, Ultragenix), and travel fees (BioMarin, Cello Health Sciences, Lumos Pharma, SigmaTau/AlphaSigma). JV has received research support (BioMarin). BKB has participated in advisory boards (BioMarin, ReGenXBio), received research support (Shire), received consulting fees (Alexion, Shire, BioMarin), and participated as trial investigator (BioMarin, Shire, Ultragenyx, Alexion Armagen, Cytonet). JAT has participated in advisory boards and received research support (BioMarin). CD, ML, KL, JJ, and KL are BioMarin Pharmaceutical Inc. employees and stockholders.
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