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Niagen Bioscience Announces First-Ever Peer-Reviewed Study Highlighting the Potential of Nicotinamide Riboside (NR) for Werner Syndrome, a Rare Genetic Disorder
Niagen Bioscience, Inc. (NASDAQ: NAGE) (formerly ChromaDex Corp.), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, shares positive results from a clinical study published in the peer-reviewed journal Aging Cell, by a team led by Masaya Koshizaka, M.D., Ph.D., Associate Professor, and Koutaro Yokote, M.D., Ph.D., MBA, President of Chiba University, both of the Center for Preventive Medical Sciences, Chiba University, and the Department of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Japan. This is the first study to demonstrate the safety and efficacy of Niagen Bioscience’s patented nicotinamide riboside (NR) ingredient, Niagen, in individuals with Werner syndrome (WS), a rare genetic disorder marked by rapid aging and premature mortality.
The newly published double-blind, placebo-controlled study found that daily supplementation with Niagen significantly elevated blood NAD+ levels by approximately 140% and improved multiple clinical markers of cardiovascular and skin health in individuals with WS. Affecting approximately 1 in 380,000 to 1 in 1,000,000 people globally (GeneReviews), Werner syndrome is caused by mutations that impair DNA repair, leading to cellular aging decades ahead of normal progression.
Dr. Koshizaka noted, “We hope our work will accelerate studies on not only WS but also other premature aging disorders and common age-related diseases—ultimately helping to extend health span and improve quality of life in both patients and the broader population.”
Developing NAD+ Therapies for Rare, Age-Related Diseases
This study builds on the growing body of clinical research demonstrating Niagen’s potential in rare, age-related diseases (see Table 1 below). In Ataxia Telangiectasia (AT), Niagen has shown improvements in neurological function, coordination, and immune markers, including in pediatric populations.
Rob Fried, CEO of Niagen Bioscience, stated, “Rare disease research is a priority for Niagen Bioscience, particularly those indications associated with accelerated aging, mitochondrial dysfunction, or NAD+ deficiency.”
The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation to NR for the treatment of AT. These designations underscore the urgent unmet need and potential therapeutic value of Niagen in rare disease populations.
Why NAD+ Matters in Rare Disease
Scientific research has shown that declining NAD+ levels can contribute to age-related decline, mitochondrial dysfunction, and impaired DNA repair. In
both Werner syndrome and AT, NAD+ deficiency appears to play a central role in disease progression (Fang et al., 2019). By restoring NAD+ levels, Niagen may help activate protective enzymes like SIRT1 and PARP1, reduce oxidative stress, and improve tissue function (Fang et al., 2016; Veenhuis et al., 2021; Presterud et al., 2023; Shoji et al., 2025; Lautrup et al., 2025).
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