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Moving Toward Precision Medicine in Duchenne Muscular Dystrophy: Where We Are and Where We’re Going

  1. Which of the following is the most precise confirmatory diagnostic test for DMD?
    Muscle biopsy
    Genetic testing
    Creatine kinase level
  2. What is an advantage of using adeno-associated virus (AAV)-mediated investigational gene therapy for the management of DMD?
    Small packaging capacity
    Pre-existing immunity in some patients
    Numerous serotypes available
  3. SRP-9001, PF-06939926, and SGT-001 are investigational gene therapies for the management of DMD. Which of the following is a key outcome measure used in the clinical trials of these gene therapies?
    North Star Ambulatory Assessment (NSAA)
    Microdystrophin expression
    Respiratory function
  4. Mike is an 8-year-old male patient with DMD. He has a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. Which of the following medications would you start Mike on?