Treatment of thoracolumbar kyphosis in patients with MPS I
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 treated with hematopoietic cell transplantation (HCT) or enzyme replacement therapy (ERT) Therefore, a procedure was organised with the aim to develop the first clinical practice guideline for the management of thoracolumbar kyphosis in MPS I patients. The eventual goal of treatment is the maintenance or improvement of quality of life.
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