Steiner, Robert D.
DeBarber, Andrea
Larson, Austin
Blanchard, Bobbi
Laurie, Shawn
Odedara, Neel
Rosengrant, Hollisa
Dutta, Rana
Funding for this research was provided by:
American Society for Pharmacology and Experimental Therapeutics
Article History
Received: 18 May 2023
Accepted: 14 September 2023
First Online: 19 December 2023
Declarations
:
: Robert D. Steiner: Employee of PreventionGenetics, part of Exact Sciences. Equity interest in Acer Therapeutics, PTC Therapeutics, and Exact Sciences. Consulting fees from Acer Therapeutics, Best Doctors/Teladoc; Leadiant, PTC Therapeutics, and Travere Therapeutics, Inc.; and honoraria from Medscape/WebMD LLC. Research funding from Alexion Pharmaceuticals, Children's Mercy Research Institute, and the Smith Lemli Opitz Foundation. Andrea DeBarber, Austin Larson, Bobbi Blanchard, Shawn Laurie, Neel Odedara, Hollisa Rosengrant: Declare that they have no competing interests. Rana Dutta: Employee, Mirum Pharmaceuticals, Inc.
: Patients and caregivers completed informed consent forms indicating they are aware that personal medical information will be disclosed to the public. Pseudonyms were used for patients and caregivers who were unwilling to disclose their identity and details of their medical information were pseudoanyonymized. This article does not contain any new studies with human participants or animals performed by any of the authors.