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ezutromid

Indications: - Duchenne muscular dystrophy (investigative agent) Mechanism of action: - utrophin modulator - may act as functional replacement for dystrophin - diminishes inflammation in calf muscles of children with Duchenne muscular dystrophy

General

metabolic agent (metabolic modifier)

References

  1. George J, Ezutromid Shows Potential in DMD. Phase II study indicates modulating utrophin may reduce muscle inflammation. MedPage Today. April 29, 2018 https://www.medpagetoday.com/meetingcoverage/aan/72591 - Muntoni F, et al Ezutromid significantly reduced muscle damage whilst maintaining utrophin in patients with Duchenne muscular dystrophy (DMD) after 24-weeks of treatment. Anerican Academy of Neurology (AAN) 2018.