The MPS Research Laboratory is dedicated to treatment of mucopolysaccharidoses, a group of inherited lysosomal storage diseases.

About the MPS Research Lab

MPS_Research_Lab_Beach_2010

The MPS Research Laboratory is part of the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center. We are a translational research laboratory specializing in enzyme replacement therapy for mucopolysaccharidosis (MPS).

Moin Vera, M.D., Ph.D., Named LDN Fellow

Dr. Moin Vera has been awarded a fellowship in the Lysosomal Disease Network. The Lysosomal Disease Network is an NIH-supported Rare Disease Clinical Research Network (U54, NINDS).

FDA Hearing on Review and Regulation of Rare Diseases Treatments

The FDA is holding a public hearing June 29 and 30 to discuss the way the Agency regulates therapies for rare disorders ("Considerations Regarding Food and Drug Administration Review and Regulation of Articles for the Treatment of Rare Diseases; Public Hearing", FDA-2010-N-0218-0001).

Per the notice:

"This hearing is intended to provide advocates for patients with
rare diseases, academics, health care providers, the pharmaceutical
industry, and other interested parties an opportunity to relate their
experience with, concerns about, and suggestions for the way FDA

MPS Research Laboratory in Australia June 2010

Patricia Dickson and Brigette Tippin of the MPS Research Lab will be presenting their research at the 11th International Symposium on MPS and Related Diseases in Adelaide, Australia.

Patricia Dickson will present orally at the scientific program on Friday, June 25, on "Gene expression alterations in cortical neurons and response to intrathecal and intravenous enzyme replacement therapy in the MPS I dog." She will also present at the family program on "Intrathecal infusion the MPS I/Hurler Scheie experience."

NORD and EURODIS Host Rare Disease Blog

The National Organization for Rare Disorders (NORD) and Rare Diseases Europe (EURORDIS) are inviting comments on their Rare Disease Blog website.

The site features blog entries by the FDA's Tim Cote, NIH's Steve Groft, NORD's Peter Saltonstall, and EURORDIS's Yann Le Cam and Terkel Anderson, among others. It is a joint effort between the U.S. and Europe's primary rare disease groups.

WORLD Abstracts and Presentations

MPS Research Lab activities will be presented at the annual WORLD Lysosomal Disease Network meeting in Miami, Florida on February 10-12.

Dr. Matthew Ellinwood of Iowa State University will present work performed in collaboration with us on treatment of the brain using intravenous and intrathecal enzyme replacement therapy for MPS I, during the afternoon session on Wednesday Feb. 10.

Legends of Golf Tournament

From the CuretheProcess.org newsletter:

Research Challenges in CNS Manifestations of Inborn Errors of Metabolism

The National Institutes of Health (Office of Rare Diseases Research, and National Institute of Neurologic Disorders and Stroke) and the Food and Drug Administration (Center for Drug Evaluation and Research, Division of Gastroenterology Products) are sponsoring a workshop on Research Challenges in CNS Manifestations of Inborn Errors of Metabolism to be held December 7 and 8 in Bethesda, Maryland.

MPS Walk/Run Los Angeles

From Tami Slawson:

The 9th Annual National MPS Society’s Walk and Run LA is October 17th 2009 is at Oak Mesa Park in La Verne, CA (click here for directions). Please join us for this worthwhile event. All proceeds raised benefit research for children with MPS/ML and related disorders. We will again be hosting the family picnic after the walk/run for all of our MPS/ML families.

You can register online here.

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