Optimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease.
Pizzamiglio C, Stefanetti RJ, McFarland R, Thomas N, Ransley G, Hugerth M, Grönberg A, Serrano SS, Elmér E, Hanna MG, Hansson MJ, Gorman GS, Pitceathly RDS.
Pizzamiglio C, et al. Among authors: serrano ss.
Brain. 2025 Jan 7;148(1):39-46. doi: 10.1093/brain/awae308.
Brain. 2025.
PMID: 39657714
Free PMC article.
Clinical Trial.