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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

key information

study id #: NCT01957059

condition: Duchenne Muscular Dystrophy

status: terminated


The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.

Regimen Selection Phase Group 2,
Regimen Selection Phase Group 3,
Treatment Phase Group 4,
Regimen Selection Phase Group 1 (COMPLETED),
Dosing Extension

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01957059

last updated: November 22, 2018