2023
DOI: 10.3390/ijms241813988
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Femoral Structure and Biomechanical Characteristics in Sanfilippo Syndrome Type-B Mice

Frederick James Ashby,
Evelyn J. Castillo,
Yan Ludwig
et al.

Abstract: Sanfilippo syndrome Type-B, also known as mucopolysaccharidosis IIIB (MPS IIIB), accounts for approximately one-third of all Sanfilippo syndrome patients and is characterized by a similar natural history as Type-A. Patients suffer from developmental regression, bone malformation, organomegaly, GI distress, and profound neurological deficits. Despite human trials of enzyme replacement therapy (ERT) (SBC-103, AX250) in MPS IIIB, there is currently no FDA approved treatment and a few palliative options. The major… Show more

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