Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Nadia Ali, PhD
With
BioMarin Pharmaceutical
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
1. Documented clinical diagnosis of MPS IV based on clinical signs and symptoms of MPS IV and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IV.
2. Patient is at least 18 years old.
3. Patient is not currently receiving enzyme replacement therapy for MPS IV.
4. Patient must provide written, informed consent prior to study participation.
You may not be if
1. Previous treatment with ERT
2. Previous hematopoietic stem-cell transplant
3. Patient has a clinically significant disease (with the exception of symptoms of Morquio), including clinically significant immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the investigator, would confound the effects of Morquio upon study variables
