Abstract:A patient with mucopolysaccharidosis type VI, on enzyme replacement therapy (galsulfase) for more than 6-years with good adherence. Urine glycosaminoglycans became stable, cardiopulmonary progression is slow, motor skills have improved, pain is controlled and no adverse reactions were reported. These support the benefits of galsulfase, as it slows disease progression.
Key Clinical MessageA patient on enzyme replacement therapy (galsulfase) for more than 6-years. Supporting the long-term benefits of galsulfase … Show more
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