Purpose

“Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs”

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson P.I.

Updates & Results

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2889197/

Purpose

“Early Versus Late Treatment of Spinal Cord Compression With Long-Term Intrathecal Enzyme Replacement Therapy in Canine Mucopolysaccharidosis Type I.”

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson P.I.

Updates & Results

http://www.ncbi.nlm.nih.gov/pubmed/20655780

Purpose

"Gene Expression Alterations in Cortical Neurons and Response to Intrathecal and Intravenous Enzyme Replacement Therapy In the Mucopolysaccharidosis I Dog”

Location

Iowa State University, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. N. Matthew Ellinwood P.I. Iowa State University. Dr. Patricia Dickson P.I. Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Updates & Results

http://mps2010.eproceedings.com.au/presentations/Friday/HallG/1400%20Dickson.pdf

Purpose

Enzyme Replacement Therapy for Mucoploysaccharidosis (MPS Type I)

Location

Harbor UCLA Research and Education Institute

Investigators

Dr. Emil Kakkis M.D., Ph.D

Updates & Results

"Enzyme Replacement Therapy for Mucoploysaccharidosis (MPS Type I)" - PDF

Purpose

To identify abnormalities in brain structure and function over time in treated and untreated MPS patients.

Location

University of Minnesota

Investigators

Dr. Chet Whitley P.I. Dr. Elsa Shapiro P.I.

Updates & Results

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

Purpose

“A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I”

Location

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center

Investigators

Dr. Patricia Dickson, P.I, Dr. Agnes Chen, M.D.

Updates & Results

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

Purpose

To conduct clinical studies of pentosan polysulfate (PPS) in patients with mucopolysaccharidoses (MPS).

Location

Icahn School of Medicine at Mount Sinai, New York

Investigators

Calogera M. Simonaro, MPh, PhD, Associate Professor of Genetics and Genomic Sciences Edward H. Schuchman, MPh, PhD, Professor of Genetics and Genomic Sciences

Updates & Results

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.

http://www.mpssociety.org/wp-content/uploads/2013/05/bene-pharnaChem-GmbH-5.15.13.pdf