['duchenne muscular dystrophy']
['ns-065/ncnp-01', 'placebo']

        Inclusion Criteria:

          -  Male ≥ 4 years and <10 years of age

          -  Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon
             53 to restore the dystrophin mRNA reading frame;

          -  Able to walk independently without assistive devices;

          -  Ability to complete the time to stand, time to run/walk and time to climb assessments;

          -  Stable dose of glucocorticoid for at least 3 months

        Exclusion Criteria:

          -  Acute illness within 4 weeks prior to the first dose of study medication;

          -  Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on
             investigation would not be exclusionary];

          -  Severe allergy or hypersensitivity to medications;

          -  Severe behavioral or cognitive problems that preclude participation in the study, in
             the opinion of the Investigator;

          -  Previous or ongoing medical condition, medical history, physical findings or
             laboratory abnormalities that could affect safety, make it unlikely that treatment and
             follow-up will be correctly completed or impair the assessment of study results, in
             the opinion of the Investigator;

          -  Patient is taking any other investigational drug currently or within 3 months prior to
             the start of study treatment; or

          -  Patient has had surgery within the 3 months prior to the first anticipated
             administration of study medication or surgery is planned for anytime during the
             duration of the study;

          -  Patient has previously participated in this study or any other study during which
             NS-065/NCNP-01 was administered.
      