Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

Inclusion Criteria

• •Documented diagnosis of DMD (severe dystrophinopathy) and clinical features consistent of typical DMD at diagnosis (i.e., documented delayed motor skills and muscle weakness by age 5 years) and who in the opinion of the Treating physician would benefit from treatment with idebenone. DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e., absent or \<5% of normal) on Western blot or immunostaining.
• •Minimum 8 years old at Prescreening.
• •PEF or FVC ≤80% and \>25% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at the Enrollment Visit.
• •Able to understand program requirements and swallow program medication.
• •Signed and dated Informed Consent Form (to be obtained at the Enrollment Visit from patient or parent/legal guardian (if applicable) prior to performing any program-specific procedures and dispensing idebenone to the patient).