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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
- 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.