Document is current

Article History

Received: 24 June 2025

Accepted: 11 October 2025

First Online: 27 October 2025

Declarations

:

: All patient interview materials were submitted to a centralized institutional review board (IRB), Sterling IRB. No IRB approval was required or sought for the clinician advice meetings. Materials for the conduct of interviews with patients received approval from Sterling IRB on 18 October 2023. All patient interviews were conducted with written and verbal consent, and all clinician advice meetings were conducted with verbal consent.

: N/A.

: J Chickering, P Wilson, M Webster, and C Glasser are employed by and hold stock/stock options in Tisento Therapeutics, Inc. B Banderas, S Berger, P Medrano, L Settel, M Brimmer are or were employed by Adelphi Values, an outcomes consultancy, and A Shields are employed by Adelphi Values, an outcomes consultancy that received payment from Tisento Therapeutics, Inc. to support the research activities presented in this manuscript. C Gwaltney is employed by Gwaltney Consulting, an outcomes consultancy that also received payment from Tisento Therapeutics, Inc. to support the research activities presented in this manuscript. A Goldstein is employed by the Children’s Hospital of Philadelphia A Karaa is a consultant for Tisento and is a Principal Investigator of the Tisento-sponsored Phase 2 MELAS clinical trial. A Larson is a consultant for Tisento, Mirum, and UCB. He performs contracted research with Tisento, Stealth, Astellas, Neuren, and Sentanyl and serves on the scientific advisory board for the United Mitochondrial Foundation. S Parikh serves on the SMAB for the CHAMP Foundation, is a speaker for the UMDF, serves on the DSMB for Tisento, receives clinical research support from the CHAMP Foundation and is employed by the Cleveland Clinic. F Scaglia received research grants from PTC Therapeutics, Astellas Pharma, Saol Therapeutics, FDA, and the NIH (U54 NS078059 and U54 NS115198). He has received consulting payments from Acer Therapeutics, Precision BioSciences, Tisento Therapeutics, and UCB. He has received research support from the Mervar Foundation and the Courage for a Cure Foundation. KD Harrington is a full-time employee of Cogstate Ltd. P Ventola is a full-time employee of Cogstate Ltd. CJ Edgar is a consultant to Cogstate and a shareholder. He has also received consulting payments from Vanqua Bio.