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Sanfilippo Syndrome clinical trials at UCSF
2 in progress, 1 open to new patients

  • A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

    open to eligible people ages 1–10

    Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how to measure these problems over time. It will particularly look at how the disease develops in young children. This is an observational study, so no experimental drug will be given. The results from this study will help us design future studies to measure whether these health problems get better when we give experimental drug for MPS IIIB.

    Oakland, California and other locations

  • A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

    Sorry, not currently recruiting here

    This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by BioMarin in this study.

    Oakland, California and other locations