Pirinen · Cell metabolism 2020 · non-randomized matched prospective intervention study · n=?

Niacin Cures Systemic NAD + Deficiency and Improves Muscle Performance in Adult-Onset Mitochondrial Myopathy.

Cited 297 times in the scientific literature.

Level 3 - non-randomized controlled study

Non-randomized prospective trial with matched control group

PubMed 32386566 · doi:10.1016/j.cmet.2020.04.008 · record verified 2026-08-29

What was done

Adult-onset mitochondrial myopathy patients and matched controls were treated with escalating doses of niacin (vitamin B3, up to 750–1,000 mg/day; NCT03973203). Patients were treated for 10 months and controls for 4 months. Investigators measured blood and muscle NAD+ levels, muscle strength, mitochondrial biogenesis markers, muscle metabolome shifts, and liver fat.

What was found

Blood NAD+ increased in all participants by up to 8-fold, and patient muscle NAD+ reached control levels. Muscle strength and mitochondrial biogenesis increased in all participants. In patients, the muscle metabolome shifted toward the control profile, and liver fat decreased by up to 50%. Anemia tendency was noted in some patients.

Why it matters

This paper provides human evidence of systemic NAD+ deficiency in adult-onset mitochondrial myopathy and shows that oral niacin can replete NAD+ pools, improve muscle performance, and reduce hepatic fat.

Limits

The abstract omits sample size, variance metrics, and exact baseline numbers. The trial was non-randomized and open-label, lacked a placebo comparator, used different treatment durations for patients and controls (10 months vs. 4 months), and identified anemia tendency as a potential adverse effect.

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