Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition

Heather L. Narver, Lingling Kong, Barrington G. Burnett, Dong W. Choe, Marta Bosch-Marcé, Addis A. Taye, Michael A. Eckhaus, Charlotte J. Sumner

Research output: Contribution to journalArticle

Abstract

Early treatment with the histone deacetylase inhibitor, trichostatin A, plus nutritional support extended median survival of spinal muscular atrophy mice by 170%. Treated mice continued to gain weight, maintained stable motor function, and retained intact neuromuscular junctions long after trichostatin A was discontinued. In many cases, ultimate decline of mice appeared to result from vascular necrosis, raising the possibility that vascular dysfunction is part of the clinical spectrum of severe spinal muscular atrophy. Early spinal muscular atrophy disease detection and treatment initiation combined with aggressive ancillary care may be integral to the optimization of histone deacetylase inhibitor treatment in human patients.

Original languageEnglish (US)
Pages (from-to)465-470
Number of pages6
JournalAnnals of neurology
Volume64
Issue number4
DOIs
StatePublished - Oct 1 2008

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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    Narver, H. L., Kong, L., Burnett, B. G., Choe, D. W., Bosch-Marcé, M., Taye, A. A., Eckhaus, M. A., & Sumner, C. J. (2008). Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition. Annals of neurology, 64(4), 465-470. https://doi.org/10.1002/ana.21449