- 1General pediatric physical examination, including anthropometric measurements (weight and height). 2Blood pressure and heart rate. 3Including visual acuity and slit-lamp examination. 4Motor function measurement, North Star Ambulatory Assessment, proximal upper limb function, timed function tests including 6-min walk test, 10-min walk/run test, supine up time, and quantitative muscle testing using grip force. 5Thigh muscle fat fraction and T2 relaxation time on quantitative magnetic resonance imaging (MRI). 6Personal-Adjustment and Role Skills Scale. 7AChemistry: creatine kinase, gamma GT, total bilirubin, alkaline phosphatase, creatinine, electrolytes (Na+, K+, Ca2+), urea, triglycerides; hematology: full blood count for erythrocytes, leukocytes (with a differential), platelets, hemoglobin, hematocrit, absolute neutrophil count. 7BSex hormone function (luteinizing hormone, follicle-stimulating hormone, testosterone, sex hormone binding globulin, alpha-fetoprotein). 8In selected sites only. *Will be assessed at visit 5 (end of study visit). d day, DEXA dual-energy x-ray absorptiometry, DVT deep vein thrombosis, TC telephone call
Trials