Review
Open access
Aug 2026
2000–2025: Emerging therapies in primary mitochondrial disease: A structured review, horizon scan, and roadmap for the future
Sustaining momentum in PMD will require more rigorous alignment of trial design with molecular biology, strengthening of natural history infrastructure, deployment of sensitive biomarkers, and adoption of innovative statistical approaches.
A. Karaa, M. I. G. Lopez Sanchez, Claire Stuart et al.
· Therapeutic Advances in Rare... · 0 citations