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P.S.K. has received research/grant support from Sanofi Genzyme, Valerion Therapeutics, Shire Pharmaceuticals, Amicus Therapeutics, Pfizer, Alexion Pharmaceuticals, and Ultragenyx, and consulting fees and honoraria from Sanofi Genzyme, Shire Pharmaceuticals, Alexion Pharmaceuticals, Amicus Therapeutics, Vertex Pharmaceuticals, Ultragenyx, and Asklepios Biopharmaceutical, Inc. (AskBio). P.S.K. is listed as an inventor on a licensed Duke University patent for the use of rhGAA in the treatment of GSDIII and other GSDs excluding GSDII. To date, neither Duke University nor the inventor has received any money from the commercialization of rights associated with this patent. Stephanie Austin received consulting fees from Ultragenyx. S.P.Y. works for a laboratory that offers clinical testing for urinary Glc4. She has received grant support from Sanofi Genzyme, Amicus Therapeutics, Biomarin Pharmaceutical, PTC Therapeutics, and Valerion Therapeutics and has consulted for Amicus Therapeutics, Sanofi Genzyme, and PTC Therapeutics. The other authors declare no conflicts of interest.
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Young, S.P., Khan, A.A., Austin, S.L. et al. Response to Heiner-Fokkema et al.. Genet Med (2020). https://doi.org/10.1038/s41436-020-0879-1