Duchenne muscular dystrophy (DMD): other treatments
Evidence-based neurology checklist on duchenne muscular dystrophy (dmd): other treatments: 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…
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
- Lamb YN. Givinostat: first approval. Drugs 2024; 84:849-856.
- 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.
- 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.
Related checklists
- Duchenne muscular dystrophy (DMD): clinical features
- Duchenne muscular dystrophy (DMD): female carriers
- Duchenne muscular dystrophy (DMD): investigations
- Duchenne muscular dystrophy (DMD): bone health management
- Duchenne muscular dystrophy (DMD): respiratory management
- Duchenne muscular dystrophy (DMD): cardiac management
- Duchenne muscular dystrophy (DMD): genetic treatments
- Duchenne muscular dystrophy (DMD): steroid management
- Duchenne muscular dystrophy (DMD): investigational treatments
