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Article History

Received: 11 October 2019

Accepted: 22 April 2020

First Online: 22 May 2020

Ethics approval and consent to participate

: Not applicable.

: Not applicable.

: JJ and RC are employees of Cure SMA and report grants from The Cure SMA Industry Collaboration which provided funding for this project. ISP, AMS, and FHS are employees of Faegre Drinker Biddle & Reath LLP. They report paid professional fees to Faegre Drinker Biddle & Reath LLP for their involvement in the design, conduct, and analysis of the work described herein, but did not receive payment for authorship of this manuscript. The support received to conduct this study was noted and it does not result in a conflict of interest for any of the authors. The authors have indicated that they have no other conflicts of interest regarding the content of this article. None of the authors has financial stake in any SMA drug or company involved in developing SMA drugs. Study PI (Dr. Jarecki) did not receive any honoraria or consultancies from industry for her involvement in this study. Study sponsor did not participate in study design, collection, data analysis and interpretation of data or writing of the manuscript. It was the decision of Cure SMA to submit this manuscript for publication.