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Case28: Female, 6 yr onset, 7 yr diagnosis, 9.9 yr report
HPOsFreeText: Wheelchair partially used beginning at 9 yr, Respiratory support used at night beginning at 8 yr, FVC z-score sitting -6.36 (30% pred), FVC z-score supine -6.88 (25% pred), 82% MRC Scale, 979 CK U/l, 0.3 GAA activity (nmol/h/mg)^h from cultured fibroblasts