Table 1

Key medical and physiotherapy information recorded on the database

Baseline informationMedical information at follow-upOutcome measures at follow-up
Demographic dataDate of starting GCsAmbulation status and mobility aids
Genetic mutation*Current dose and regimen of GCsAge at loss of independent ambulation
Maternal carrier status*Adverse behavioural changesRespiratory status (FVC, FVC%)
Date of diagnosisGastrointestinal symptomsEchocardiogram (LVFS%)
Features of the muscle biopsy†Increased appetiteNorthStar Ambulatory Assessment score
Family and social historyHistory of infectionsTime rising from the floor from lying
Height and weightTimed 10 m run
Blood pressureManual muscle testing
Cushingoid featuresJoint range
Bone density measurementsSpinal posture
Long bone fractures and vertebral fractures
Delayed puberty
Other therapeutic interventions
Adjustment in GC dose/regimen
  • *Genetic diagnosis and maternal carrier status confirmed by a state-of-the-art DNA diagnostic technique covering all Duchenne muscular dystrophy gene exons.

  • †Dystrophin expression observed on muscle biopsy by immunohistochemistry with monoclonal antibodies dys1, dys2, dys3 (ie, complete absence, traces).

  • FVC%, forced vital capacity percentage; GC, glucocorticoid corticosteroids; LVSF%, left ventricular shortening fraction percentage.