Patient case (clinical description): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient's own account: I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Clinical trial: Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

Eligibility criteria:
Inclusion Criteria:

          1. Patients 10 - 18 years of age at Baseline.

          2. Signed and dated informed consent.

          3. Documented diagnosis of DMD or severe dystrophinopathy and clinical features
             consistent of typical DMD at diagnosis (i.e. documented delayed motor skills and
             muscle weakness by age 5 years). 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 immunostain.

          4. Ability to provide reliable and reproducible repeat PEF within 15% of the first
             assessment (i.e. Baseline vs. Screening).

          5. Patients assessed by the investigator as willing and able to comply with the
             requirements of the study, possess the required cognitive abilities and are able to
             swallow study medication.

        Exclusion Criteria:

          1. Patients dependent on assisted ventilation at Screening and/or Baseline (defined as
             non-invasive nocturnal ventilation, daytime non-invasive ventilation or continuous
             invasive ventilation).

          2. Patients with documented DMD-related hypoventilation for which assisted ventilation is
             needed according to current standard of care guidelines (e.g. FVC< 30%) or is required
             in the opinion of the Investigator.

          3. Patients with a percent predicted PEF > 80% at Baseline.

          4. Patients unable to form a mouth seal to allow precise respiratory flow measurements
             and mouth pressures.

          5. Symptomatic heart failure (high probability of death within one year of Baseline)
             and/or symptomatic ventricular arrhythmias.

          6. Participation in the previous Phase II or Phase II Extension study (SNT-II-001 or
             SNT-II-001-E) for idebenone.

          7. Participation in any other therapeutic trial and/or intake of any investigational drug
             within 90 days prior to Baseline.

          8. Use of carnitine, creatine, glutamine, oxatomide, or any herbal medicines within 30
             days prior to Baseline.

          9. Use of coenzyme Q10 or vitamin E (if taken at a dose of 5 times above the daily
             physiological requirement) within 30 days prior to Baseline.

         10. Any previous use of idebenone.

         11. Any concomitant medication with a depressive or stimulating effect on respiration or
             the respiratory tract.

         12. Planned or expected spinal fixation surgery during the study period (as judged by the
             investigator).

         13. Asthma, bronchitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any
             other non-DMD respiratory illness that affects PEF.

         14. Chronic use of beta-2 agonists or any use of other bronchodilating medication (e.g.
             inhaled steroids, sympathomimetics, anticholinergics).

             Please note: Chronic use if defined as a daily intake for more than 14 days.

         15. Moderate or severe hepatic impairment or severe renal impairment.

         16. Prior or ongoing medical condition or laboratory abnormality that in the
             Investigator's opinion could adversely affect the safety of the subject.

             Please note: Patients who suffer from a severe, unstable condition including (but not
             limited to) cancer, auto-immune diseases, haematological diseases, metabolic disorders
             or immunodeficiencies, and who are at risk of an aggravation unrelated to the study
             condition, can only be included in the study if accepted in writing by the Sponsor's
             Medical Monitor.

         17. Relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana
             products/smoking

         18. Known individual hypersensitivity to idebenone or to any of the ingredients/excipients
             of the study medication

         19. Systemic glucocorticoid therapy

               1. Chronic use of systemic glucocorticoid therapy for DMD related conditions within
                  12 months of Baseline (the "12 month non-use period")

               2. More than 2 rounds of acute systemic glucocorticoid burst therapy (of ≤2 week
                  duration) for non-DMD related conditions within the 12 month non-use period

               3. Use of any round of systemic glucocorticoid burst therapy of longer than 2 weeks
                  duration within the 12 month non-use period

               4. Use of systemic glucocorticoid burst therapy less than 8 weeks prior to baseline
Male
No healthy subjects accepted to join the trial.
Subject must be at least 10 Years old.
Subject must be at most 18 Years

Eligibility (NLI label): Contradiction