The natural history study of patients with Sanfilippo disease(s) (MPS3)
This is a natural history study of patients with Sanfilippo Disease (MPS3). Patients will be followed over the course of 6 months in which they have blood and urine collected, hearing assessment, complete questionnaires and are evaluated by the Principal Investigator.
Study Type
OBSERVATIONAL
Enrollment
6
LDRTC
Fairfax, Virginia, United States
RECRUITINGTo characterize the disease natural history in patients with MPS3.
To characterize the disease natural history in patients with MPS3.
Time frame: 6 months
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