"Enzyme Replacement Therapy for Mucoploysaccharidosis (MPS Type I)" - PDF
To identify abnormalities in brain structure and function over time in treated and untreated MPS patients. To identify distinct neuroimaging and neuropsychological patterns for each MPS disorder. To develop quantitative measurements of change. To examine the degree to which risk factors influence brain and Quality of Life outcomes.
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This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I).
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Dr. Simonaro and Dr. Schuchman have demonstrated in MPS animal models that PPS has the effect of reducing the chronic inflammation associated with substrate accumulation, resulting in improved motility and a slowing of bone/cartilage disease.
The Ryan Foundation provides funding to a wide range of research institutions and facilities in the pursuit of treatments for rare disease. These include:
UNIVERSITY OF MINNESOTA
IOWA STATE UNIVERSITY
UNIVERSITY OF PENNSYLVANIA
WASHINGTON UNIVERSITY / ST. LOUIS
UNIVERSITY OF UTAH