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Authors
Zilber, Sophia
Burnworth, Melinda
Afolabi, Titilola
Brestoff, Jonathan R.
Minczuk, Michal
Luis, Alejandro Rodriguez
Ling, Qinglan
Prigione, Alessandro
Tolle, Isabella
Elsharkawi, Ibrahim
Perlstein, Ethan
Boyce, Danielle
Johnson, Simon
Woleben, Kasey
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Article History
Received: 15 August 2025
Accepted: 27 October 2025
First Online: 20 November 2025
Declarations
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: The Leigh Syndrome Global Patient Registry study was conducted in accordance with the Declaration of Helsinki and approved by the Institutional Review Board of Sanford Health (03-13-060). Informed consent was obtained from all subjects involved in the study.
: Not applicable.
: AP has filed patent applications for the use of sildenafil for the treatment of complex IV and complex V defects. A.P. and Cure Mito have filed patent applications for the use of azole compounds talarozole and sertaconazole for the treatment of Leigh syndrome.
