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Authors
Saby, Joni N.
Peters, Sarika U.
Benke, Timothy A.
Standridge, Shannon M.
Swanson, Lindsay C.
Lieberman, David N.
Olson, Heather E.
Key, Alexandra P.
Percy, Alan K.
Neul, Jeffrey L.
Nelson, Charles A.
Roberts, Timothy P. L.
Marsh, Eric D. https://orcid.org/0000-0003-3264-0902
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Funding
Funding for this research was provided by:
National Institute of Child Health and Human Development (U54HD061222)
Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania
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Article History
Received: 26 October 2022
Accepted: 23 February 2023
First Online: 4 March 2023
Declarations
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: This work was approved by the appropriate Institutional Review Boards of CHOP, VUMC, BCH, CCH, and UC-CHCO. For the umbrella Natural History protocol (5211) the appropriate Institutional Review Boards of CHOP and VUMC approved the protocol, whereas UC-CHCO, BCH, and CCH relied on the single-IRB agreement provided by the University of Alabama at Birmingham. Written informed consent was obtained for each participant according to the Declaration of Helsinki. Data acquisition occurred between 2017 and 2021. A certificate of Confidentiality was provided by the National Institute of Child Health and Development (NICHD). This non-interventional clinical trial protocol was registered with Clinical Trials.Gov (NCT02738281).
: All authors have provided approval of the manuscript. This work has not been published in any form elsewhere.
: No author has competing interests directly related to this work.The following authors have no competing interests: J.N.S., S.U.P, S.M.S, L.C.S, D.N.L, A.P.K, C.A.N.,T.RThe following authors disclose the following competing interests:H.E.O- Site PI for clinical trials sponsored by Ovid Therapeutics and Marinus.Pharmaceuticals. Consulting for Takeda, Ovid Therapeutics, and Zogenix.J.L.N- Site PI for clinical trials with Acadia; Consulting for Acadia Pharmaceuticals, Analysis Group, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Myrtelle, Neurogene, Newron Pharmaceuticals, Signant Health, Taysha Gene Therapies, and the preparation of CME activities for PeerView Institute and Medscape; serves on the scientific advisory board of Alcyone Lifesciences; is a scientific cofounder of LizarBio Therapeutics; and was a member of a data safety monitoring board for clinical trials conducted by Ovid Therapeutics.T.A.B- Site PI for clinical trials with Acadia, GW, Takeda, and Marinus; Consulting for Acadia Pharmaceuticals.A.K.P- Site PI for clinical trials with Acadia; Consulting for Acadia Pharmaceuticals.E.D.M- Site PI for clinical trials with Acadia, Marinus, Takada, Stoke Pharmaceuticals. Consulting for Acadia Pharmacuticals.
