Deng, Sheng
Feely, Shawna M. E.
Shi, Yong
Zhai, Hong
Zhan, Luna
Siddique, Teepu
Deng, Han-Xiang http://orcid.org/0000-0002-0030-8465
Shy, Michael E.
Funding for this research was provided by:
Foundation for the National Institutes of Health (NS078287, NS099623, U54NS065712)
Article History
Received: 21 June 2019
Accepted: 20 August 2019
First Online: 29 August 2019
Compliance with Ethical Standards
:
: Dr. Michael E. Shy is a consultant for Alnylam, Inflectis, and Acceleron. The other authors declare no competing financial interests.
: Ethical approval was obtained from the University of Iowa (Iowa City, IA) and University of Wisconsin (UW; Madison, WI), and written informed assent/consent was provided by participants under a protocol approved by the ethics board of the NIH Rare Diseases Clinical Research Network (Protocol INC6611).
: Written informed consent was obtained from all participants involved in this study.