Author

A. Lim

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Review Open access Jul 2026

Advances in Primary Mitochondrial Diseases: Diagnosis, Natural History Studies and Clinical Trials

Advances in therapeutic discoveries and clinical trials are challenging the obsolete dogma that PMDs are untreatable and bringing hope to patients; four compounds have been licensed, and many trials are in progress.

A. Lim, Aye Moe, R. Stefanetti et al. · Genes · 0 citations