Nicotinamide Riboside Supplementation Benefits in Patients With Werner Syndrome: A Double-Blind Randomized Crossover Placebo-Controlled Trial

Objective

To evaluate the safety and efficacy of oral NR supplementation in patients with Werner syndrome (WS).

Study Design

Randomized, double-blind, placebo-controlled, crossover study in 9 WS patients

Dose

1000 mg

Duration

52 weeks

Key Outcomes

  • NR improved cardio-ankle vascular index (CAVI), a measure of arterial stiffness.

  • NR increased the number of large HDL particles, indicating potential cardiovascular benefits.

  • NR reduced skin ulcer size and heel pad thinning, while ulcers worsened in the placebo group.

  • Although mild liver enzyme elevations were noted, they were deemed manageable and consistent with underlying liver sensitivities common in Werner syndrome.

Clinical, Orphan DiseaseANAD Staff