Tier 4 — mechanistic

Effects of nicotinamide riboside in mitochondrial DNA disease

Katherine R. Schon, Alison Sleigh, Zoe Golder, Alessandro Berghella, May Yung Tiet, Camilla Lyons, Lyuba V. Bozhilova, Yu Nie, Emma Harrison, Chloe Seikus, Patrick Yu-Wai-Man, Annelies Quaegebeur, Andreas Roos, Andreas Hentschel, Heather Biggs, Massimo Zeviani, Carlo Viscomi, Jelle van den Ameele, Rita Horvath, Patrick F. Chinnery
Genome Medicine 2026

Bibliography

DOI
10.1186/s13073-026-01764-1
Funding
UK and international public, university and charitable support, including MRC, NIHR, Wellcome and rare-disease programs; Niagen Bioscience supplied NR.
Competing interests
The authors reported no relevant conflicts and disclosed institutional and editorial roles.

Study snapshot

DesignOpen-label human intervention study
ModelHuman participants
Sample8 adults; 3 mtDNA deletion and 5 m.3243A>G/T cases
InterventionNR 1250–2000 mg/day
Duration4 weeks
EndpointsTimed up and go; muscle respiratory-chain measures; mtDNA

What the study showed, in plain terms

The experimental-medicine study found NR bioavailability, a timed-up-and-go signal and genotype-dependent muscle findings.

Key findings

The experimental-medicine study found NR bioavailability, a timed-up-and-go signal and genotype-dependent muscle findings.

What this study can and cannot tell us

Eight participants without a placebo group; favorable muscle changes were concentrated in the deletion subgroup. The accepted article is not proof of a broadly effective mitochondrial-disease treatment.

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Editorial review

Reviewed by the Biohack Blueprint research team

Last verified

Dr. Khaled Mahmoud, MBBChCritical Care Medicine, Longevity Science & Editorial Direction Dr. Amr Yasser, PhDCardiometabolic, Musculoskeletal, Gut & Bioavailability Dr. Abdulla Mostafa, PhDCellular Senescence, Neurological, Mitochondrial & Brain-Ageing Biology