Duchenne muscular dystrophy (DMD): other treatments

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

Duchenne muscular dystrophy (DMD): other treatments. This is a histone deacetylase inhibitor. Fully referenced neurology checklist.

Givonistat

  • This is a histone deacetylase inhibitor
  • It has been approved for patients at least 6 years old
  • It is administered orally twice a day with food
  • The most frequent adverse events are diarrhoea and vomiting
  • It increases muscle tissue and reduces fibrotic tissue
  • It also reduces tissue necrosis and fatty replacement
  • Givonistat is also used in the treatment of polycythemia vera

References

  1. Lamb YN. Givinostat: first approval. Drugs 2024; 84:849-856.
  2. Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol 2024; 23:393-403.
  3. Bettica P, Petrini S, D'Oria V, et al. Histological effects of givinostat in boys with Duchenne muscular dystrophy. Neuromuscul Disord 2016; 26:643-649.

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