Nicotinamide Riboside Improves Ataxia Scores and Immunoglobulin Levels in Ataxia Telangiectasia

Objective

To investigate the effects of NR supplementation in patients with ataxia telangiectasia (AT).

Study Design

Open-label proof-of-concept study in 24 patients with AT

Dose

25 mg/kg

Duration

4 months

Key Outcomes

  • NR supplementation improved ataxia scores (SARA and ICARS). However, this improvement disappeared after NR withdrawal, indicating a temporary, symptomatic effect of NR in AT.

  • NR also markedly increased serum immunoglobin G (IgG) in immunodeficient patients.

  • This is the first clinical study to investigate the effects of NR in patients with AT, and most notably, the first clinical NR trial in children under the age of 18.

Clinical, Ataxia Telangiectasia, Neurological HealthANAD Staff