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Authors
Inoue, Michio
Jayaraman, Divya
Bengoechea, Rocio
Bhadra, Ankan
Genetti, Casie A.
Aldeeri, Abdulrahman A.
Turan, Betül
Pacheco-Orozco, Rafael Adrian
Al-Maawali, Almundher
Al Hashmi, Nadia
Zamani, Ayşe Gül
Göktaş, Emine
Pekcan, Sevgi
Çağlar, Hanife Tuğçe
True, Heather
Beggs, Alan H.
Weihl, Conrad C.
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Funding
Funding for this research was provided by:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (K99AR084062, R01AR068797, R01AR068797)
Muscular Dystrophy Association (MDA961862)
National Institute of Neurological Disorders and Stroke (R25NS070682)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (P50HD105351)
the Boston Children’s Hospital CRDC Initiative
Lee and Penny Anderson Family Foundation
Jonathan and Deborah Parker for the Giving Strength Foundation
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Article History
Received: 14 August 2024
Accepted: 18 October 2024
First Online: 28 October 2024
Declarations
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: Written informed consent for publication was obtained from the patients or their parents.
: The authors declare no competing interests.
: The medical history, clinical evaluation, and human samples used in this study were obtained for diagnostic purposes. This study was approved by the Institutional Review Board of Washington University School of Medicine (IRB #201103416) and Boston Children’s Hospital (protocol 03-08-128R).
