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

Ann Neurol. 2008 Oct;64(4):465-70. doi: 10.1002/ana.21449.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age Factors
  • Animals
  • Animals, Newborn
  • Body Weight / drug effects
  • Body Weight / physiology
  • Disease Models, Animal
  • Disease Progression
  • Enzyme Inhibitors / therapeutic use*
  • Hydroxamic Acids / therapeutic use*
  • Mice
  • Mice, Transgenic
  • Motor Activity / drug effects
  • Muscular Atrophy, Spinal / therapy*
  • Necrosis
  • Nutritional Support / methods*
  • Survival Analysis
  • Survival of Motor Neuron 1 Protein / genetics


  • Enzyme Inhibitors
  • Hydroxamic Acids
  • Survival of Motor Neuron 1 Protein
  • trichostatin A