The dataset provides an extensive examination of the protein and RNA expression profiles of dystrophin isoforms in the brain, utilizing Western Blot and quantitative Polymerase Chain Reaction (qPCR) a
Purpose SMT C1100 is a utrophin modulator being evaluated as a treatment for Duchenne muscular dystrophy (DMD). This study, the first in pediatric DMD patients, reports the safety, tolerability and PK
