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).
More Information can be found at:
http://clinicaltrials.gov/show/NCT00852358
http://mpsresearchlab.labiomed.org/joomla2/index.php/our-research/14-funding/18-ryan-foundation
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.
For more information:
https://events-support.com/Documents/Shapiro.pdf
http://www.mps2012.eu/upload/siteimages/MPS2012/S04.3.pdf
http://www.ag.iastate.edu/news/releases/903/
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3075726/
http://harborpeds.org/news/pattidicksonagneschenMPSpaper2010
http://stm.sciencemag.org/content/2/60/60ra89.abstract
The Ryan Foundation provides funding to a wide range of research institutions and facilities in the pursuit of treatments for rare disease. These include:
UCLA
UNIVERSITY OF MINNESOTA
IOWA STATE UNIVERSITY
UNIVERSITY OF PENNSYLVANIA
WASHINGTON UNIVERSITY / ST. LOUIS
UNIVERSITY OF UTAH