Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here?
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- Record sourced from PubMed, PMID 42229401.
- Also identified by DOI 10.1016/j.cmet.2026.04.014.
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Abstract
After decades without approved pharmacotherapies, mitochondrial disease care is shifting. Two FDA approvals emerged in 1 year, elamipretide (Forzinity) for Barth syndrome and deoxynucleoside therapy (Kygevvi) for TK2 deficiency, with another under review. Zink et al.<sup>1</sup> suggest sildenafil (Viagra) could treat Leigh syndrome, highlighting drug repurposing for severe pediatric mitochondrial disease.