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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
study id #: NCT01957059
condition: Duchenne Muscular Dystrophy
status: terminated
purpose: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.
intervention:
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
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