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ezutromid (SMT C1100) (formerly BMN 195)

Also known as: formerly VOX C1100, VOXC 1100

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Company Summit Therapeutics plc
DescriptionSmall molecule utrophin (UTRN) modulator
Molecular Target Utrophin (UTRN)
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation U.S. - Fast Track (Treat Duchenne muscular dystrophy (DMD));
U.S. - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
U.S. - Rare Pediatric Disease (Treat Duchenne muscular dystrophy (DMD));
EU - Orphan Drug (Treat Duchenne muscular dystrophy (DMD))
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