Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure
Abstract Background In all patients with mucopolysaccharidosis type I (MPS I), skeletal disease (dysostosis multiplex) is a prominent, debilitating, condition related complication that may impact strongly on activities of daily living.Unfortunately, it is not alleviated by treatment with hematopoietic cell transplantation (HCT) or enzyme replacemen