MPS Research Lab

The MPS Research Lab has joined the UCLA Neuroscience Interdepartmental Program. The IDP is a graduate (Ph.D.) program accepting 15 students per year to study all aspects of neuroscience (from cellular mechanisms to disease and translation). A full faculty list can be found here.

A new study on atherosclerotic-like changes in the arterial vasculature due toMPS I and the effects of enzyme replacement therapy has made the cover of the May 2011 edition of the journal Laboratory Investigation. The authors, at the University of Tennessee, analyzed the aortas from studies performed at Iowa State University and the MPS Research Lab at LA BioMed-Harbor-UCLA.

The MPS Research laboratory participated in a landmark study published today in Science Translational Medicine. High doses of enzyme replacement therapy from birth prevented lysosomal storage in most areas of the body in a preclinical model. The work, performed also at Iowa State University by Matthew Ellinwood and Ashley Dierenfeld, shows that newborn screening may be the single most effective tool against MPS I.

Read more at http://stm.sciencemag.org/content/2/60/60ra89.abstract

Former MPS Research Lab principal investigator Emil Kakkis, M.D., Ph.D., will be honored as a Rock Star of Science in December's GQ Magazine. Dr. Kakkis is being honored for his many contributions scientifically and in public policy for individuals with rare diseases.

See http://www.crdnetwork.org/blog/2010-rock-stars-of-science%E2%84%A2-dr-em...

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).

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

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."

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.

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.