Effect of the Butyrate Prodrug Pivaloyloxymethyl Butyrate (AN9) on a Mouse Model for Spinal Muscular Atrophy.

Authors

Jonathan D. Edwards, Ohio State University Wexner Medical Center
Matthew E.R. Butchbach, Ohio State University Wexner Medical Center; Nemours Alfred I. DuPont Hospital for Children; Thomas Jefferson University; University of DelawareFollow

Document Type

Article

Publication Date

11-29-2016

Comments

This article has been peer reviewed. It is the authors' final version prior to publication in Journal of Neuromuscular Diseases, Volume 3, Issue 4, November 2016, Pages 511-515.

The published version is available at https://doi.org/10.3233/JND-160187. Copyright © IOS Press and the authors

Abstract

Spinal muscular atrophy (SMA) is an early-onset motor neuron disease that leads to loss of muscle function. Butyrate (BA)-based compounds markedly improve the survival and motor phenotype of SMA mice. In this study, we examine the protective effects of the BA prodrug pivaloyloxymethyl butyrate (AN9) on the survival of SMNΔ7 SMA mice. Oral administration of AN9 beginning at PND04 almost doubled the average lifespan of SMNΔ7 SMA mice. AN9 treatment also increased the growth rate of SMNΔ7 SMA mice when compared to vehicle-treated SMNΔ7 SMA mice. In conclusion, BA prodrugs like AN9 have ameliorative effects on SMNΔ7 SMA mice.

Recommended Citation

Edwards, Jonathan D. and Butchbach, Matthew E.R., "Effect of the Butyrate Prodrug Pivaloyloxymethyl Butyrate (AN9) on a Mouse Model for Spinal Muscular Atrophy." (2016). Department of Pediatrics Faculty Papers. Paper 81.
https://jdc.jefferson.edu/pedsfp/81

PubMed ID

27911337

Language

English