This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for ≥9 months. The study will have a single visit (Visit 1).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
6
Ataluren will be administered as per the dose and schedule specified in the arm.
University of California, Los Angeles (UCLA)
Los Angeles, California, United States
Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL)
The mean dystrophin protein levels were measured by ECL. Dystrophin levels are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). Results below the limit of quantitation were imputed as half of lower limit of quantitation (LLOQ). LLOQ = 0.5 micrograms (μg)/milliliter (mL)
Time frame: Day 1 of biopsy
Dystrophin Protein Levels as Determined by Immunohistochemistry
Dystrophin levels by IHC mean membrane stain density are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations).
Time frame: Day 1 of biopsy
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