Conclusions
Case report of a patient who has remained on enzyme replacement therapy
for more than 6 years, with adequate adherence to the pharmacological
treatment and no adverse reactions. GAG levels in the urine have
remained stable. The course of the disease has been slow in terms of
cardiac and pulmonary involvement, while observed improvements in motor
skills and pain control have enabled the patient to continue in school.
These findings support the long-term benefits of slowing the progression
of the disease with galsulfase-based ERT as an option to modify the
natural history of the disease and the quality of life in patients with
MPS VI.