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While Phase II trials in heart failure and primary mitochondrial myopathy did not meet primary efficacy endpoints, the TAZPOWER trial in Barth syndrome demonstrated significant improvements in muscle strength and sixminute walk distance, leading to FDA accelerated approval in 2025 [3] [10]

(PubMed) Fratantonio D, Speciale A, Molonia MS, et al

Slavich GM, Irwin MR (2014)