Nicotinamide Riboside Improves Ataxia Scores and Immunoglobulin Levels in Ataxia Telangiectasia.

ABSTRACT:

Background

Treatment of animal models with ataxia telangiectasia (A-T) with nicotinamide riboside (NR) improved their neurological outcome and survival.

Objective

The aim of this study is to investigate the effects of NR in patients with A-T.

Methods

In this open-label, proof-of-concept study, 24 patients with A-T were treated with NR during four consecutive months. The effects of NR on ataxia, dysarthria, quality of life, and laboratory parameters were analyzed.

Results

During treatment, ataxia scores improved; mean total Scale for the Assessment and Rating of Ataxia and International Cooperative Ataxia Rating Scale scores decreased to 2.4 and 10.1 points, respectively. After NR withdrawal, ataxia scores worsened. In immunodeficient patients, the mean serum Ig

SUBMITTER: Veenhuis SJG 

PROVIDER: S-EPMC9291897 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature