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 10 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).

Exclusion Criteria:

- Eligible for and able to participate in an ongoing clinical trial of idebenone.

- Is at high-risk of a fatal outcome from lung infection and/or advanced cardiomyopathy
in the opinion of the Treating physician.

- Known moderate or severe impairment of hepatic function or severe impairment of renal
function.

- Prior or ongoing medical condition or laboratory abnormality which in the Treating
physician's opinion may put the patient at significant risk or may interfere
significantly with the patient's participation in the program.

- Abuse of drugs or alcohol, which in Treating physician's opinion would interfere with
the compliance to treatment.

- Known individual hypersensitivity to idebenone or to any of the ingredients/excipients
of the program medication.