Spinal muscular atrophy (SMA): investigational treatments

Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .

Spinal muscular atrophy (SMA): investigational treatments. Phenylbutyrate, Sodium butyrate. Fully referenced neurology checklist.

Histone deacylase (HDAC) inhibitors

  • Phenylbutyrate
  • Sodium butyrate
  • Suberoylanilide hydroxamic acid
  • Trichostatin A
  • Valproic acid

Other investigational treatments

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References

  1. Lorson CL, Rindt H, Shababi M. Spinal muscular atrophy: mechanisms and therapeutic strategies. Hum Mol Genet 2010; 19:R111-R118. 
  2. Sproule DM, Kaufmann P. Therapeutic developments in spinal muscular atrophy. Ther Adv Neurol Disord 2010; 3:173-185.
  3. Burnett BG, Crawford TO, Sumner CJ. Emerging treatment options for spinal muscular atrophy. Curr Treat Options Neurol 2009; 11:90-101.
  4. Bertini E, Dessaud E, Mercuri E, et al; Olesoxime SMA Phase 2 Study Investigators. Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet Neurol 2017; 16:513-522.

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