Evaluation of range of motion showed that there were improvements in shoulder flexion, elbow extension, and knee extension that increased with time over 104 weeks. Sleep apnea declined 61% by 26 weeks, and the three patients with the most clinically severe sleep apnea all improved. NYHA classifications improved at least one class in all patients by 52 weeks. Visual acuity improved in the three patients with the worst vision. Height and weight growth velocity increased 85 and 131%, respectively, in the six prepubertal patients.
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