Akhtar Ali, S.
Kang, H.
Olney, R.
Ramos-Platt, L.
Ryabets-Lienhard, A.
Cheung, C.
Georgia, S.
Pitukcheewanont, P.
Funding for this research was provided by:
Parent Project Muscular Dystrophy
The Growth and Bone Foundation at Children’s Hospital Los Angeles
The Skirball Fellowship Research Award at the Children’s Hospital Los Angeles
Article History
Received: 29 April 2019
Accepted: 26 June 2019
First Online: 7 August 2019
Compliance with ethical standards
:
: Pisit Pitukcheewanont had research funding from Ultragenyx, Amgen, and Shire; salary from Ascendis Pharma. Anna Ryabets-Lienhard has research funding from Ultragenyx, Amgen, and Shire. The remaining authors have nothing to disclose.
: All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee. Informed consent was obtained for all subjects.