Response to de Vries et al.

Financiación

P.S.K. has received research/grant support from Sanofi Genzyme, Valerion Therapeutics, Shire Pharmaceuticals, Alexion, the National Institutes of Health, the Food and Drug Administration, and the Patient-Centered Outcomes Research Institute. She also receives consulting fees and honoraria from the Sanofi Genzyme, Shire Pharmaceuticals, Alexion Pharmaceuticals, Amicus Therapeutics and Roche. She serves as a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, and is a Registry Board Member for Alexion Pharmaceuticals. She also serves on the Data Safety Monitoring Board for PTC Therapeutics. S.R. is an employee of Sanofi and owns stock in Sanofi. The other authors declare no conflict of interest.