Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
This is a multicenter, open-label, dose-escalation study to evaluate the safety,
 tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12
 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD
 with a deletion mutation amenable to exon 51 skipping.
Inclusion Criteria:

 -  Male between 6 months to 48 months of age (inclusive)

 -  Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping

 -  Parent(s) or legal guardian(s) who is willing to provide written informed consent

 Exclusion Criteria:

 -  Received treatment that might have an effect on muscle strength or function within 12
 weeks prior to dosing

 -  Received previous or current treatment with any experimental treatment

 -  Clinically significant illness other than DMD

 -  Clinically significant laboratory abnormality

 -  Any other condition that could interfere with the patient's participation