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: Pentoxifylline in Duchenne Muscular Dystrophy

Eligibility criteria:
Inclusion Criteria:

          1. Male

          2. Age 4 to 7 years

          3. Ambulant independently. Subjects may use a wheelchair occasionally, but only for long
             distances

          4. Diagnosis of DMD confirmed by at least one of the following:

               -  Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
                  deficiency, and clinical picture consistent with typical DMD OR

               -  Gene deletion test positive (missing one or more exons) in the central rod domain
                  (exons 25-60) of dystrophin, where reading frame can be predicted as
                  'out-of-frame',

               -  and clinical picture consistent with typical DMD.

               -  Complete dystrophin gene sequencing showing an alteration (point mutation,
                  duplication, or other mutation resulting in a stop codon mutation) that can be
                  definitely associated with DMD, with a typical clinical picture of DMD.

          5. Positive family history of DMD confirmed by one of the criteria listed above in a
             sibling or maternal uncle, and clinical picture typical of DMD.

          6. Glucocorticosteroid - naïve (i.e. has not been treated with prednisone or Deflazacort
             within 1 year before onset of the study)

          7. Has not participated in other therapeutic research protocol within the last 6 months.

          8. Evidence of muscle weakness by MRC score or clinical functional evaluation

          9. Ability to provide reproducible repeat QMT bicep score of either the right or left arm
             within 15% of first assessment score.

        Exclusion Criteria:

          1. Symptomatic DMD carrier

          2. Use of any medication, nutritional supplement or herb for treatment of DMD within the
             last 3 months.

          3. Symptomatic cardiomyopathy or ventricular arrhythmias

          4. History of significant concomitant illness, impairment of blood clotting ability (as
             evidenced by increased PT/PTT or bleeding time over the upper limit of normal (ULN)),
             recent cerebral or retinal hemorrhage, bleeding diathesis, gastric ulcer, hypotension
             or significant impairment of renal or hepatic function (defined as serum creatinine
             and GGT respectively, greater than 1.5 times normal upper limit for age and gender).
Male
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.
Subject must be at most 7 Years

Eligibility (NLI label): Contradiction