Exploratory Study of NS-089/NCNP-02 in DMD

Inclusion Criteria

• •Has an out of frame deletion(s) that could be corrected by skipping exon 44 as confirmed by any of methodology at the time of visit 1. If not confirmed by any of methodology that evaluates the relative copy number of all exons (i.e. MLPA etc), must be confirmed through these techniques by the time of visit 3.
• •DNA sequencing of exon 44 confirms that no DNA polymorphisms occur that could compromise duplex formation between NS-089/NCNP-02 and pre-mRNA.
• •Male and \>= 8 years and \< 17 years of age at the time of obtaining informed consent and/or assent. Subjects aged \>= 4 years and \< 8 years can be enrolled according to the circumstances.
• •Able to give informed consent in writing signed by parent(s) or legal guardian who is able to understand all of the study procedure requirements. If applicable, able to give informed assent in writing signed by the subject.
• •Life expectancy of at least 1 year
• •Able to ambulate. Non-ambulant subject can be enrolled according to the circumstances.
• •Have intact muscles, which have adequate quality for biopsy. (No lacks or severe atrophy of biceps brachii or tibialis anterior muscle)
• •QTc \<450 msec (based on 12-lead ECGs), or \<480 msec for subject with Bundle Branch Block.
• •Glucocorticoid-naive patients, or patients who have used systemic glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.