Wang, Jason
Zhou, Chris J.
Khodabukus, Alastair
Tran, Sabrina
Han, Sang-Oh
Carlson, Aaron L.
Madden, Lauran
Kishnani, Priya S. http://orcid.org/0000-0001-8251-909X
Koeberl, Dwight D.
Bursac, Nenad
Article History
Received: 28 July 2020
Accepted: 31 March 2021
First Online: 5 May 2021
Competing interests
: P.S.K. has received research/grant support from Sanofi Genzyme, Valerion Therapeutics, and Amicus Therapeutics. P.S.K. has received consulting fees and honoraria from Sanofi Genzyme, Amicus Therapeutics, Vertex Pharmaceuticals, and Asklepios Biopharmaceutical, Inc. (AskBio). P.S.K. is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Amicus Therapeutics, and Baebies. D.D.K. has received research/grant support from Sanofi Genzyme, Sangamo Therapeutics, Pharming, Viking Therapeutics, and Amicus Therapeutics. D.D.K. has received consulting fees and honoraria from Sanofi Genzyme, Amicus Therapeutics, Sangamo Therapeutics, Vertex Pharmaceuticals and Asklepios Biopharmaceutical, Inc. (AskBio). D.D.K. and P.S.K. have equity in AskBio, which is developing gene therapies for Pompe disease. D.D.K., P.S.K., and Duke University might benefit financially if the experimental treatments discussed here prove effective and are successful commercially. All other authors have no competing interests.