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NIH grant for the move toward clinical trials targeting the lysosomal storage disease MPSIIIB
Date:5/25/2011

efore a clinically relevant approach," said Dr. Fu.

"The Center for Gene Therapy at Nationwide Children's Hospital has been a leader in bridging gene therapy trials from the bench into the clinic, and we are excited about bringing that expertise to bear on this devastating disorder," added Dr. Flanigan.

Dr. Fu's MPS IIIB gene therapy project has received generous support since 2003 from the MPS III patient community through Ben's Dream The Sanfilippo Research Foundation. A research grant from the foundation was critical in supporting Dr. Fu's team while they were pursuing the NIH grant application.

"We believe the quote that 'Hope sees the invisible, feels the intangible and achieves the impossible,'" said Jennifer Siedman, president and secretary of Ben's Dream  the Sanfilippo Research Foundation. "Dr. Fu's recent research breakthrough and its recognition by the NIH with the awarding a U01 grant, brings to the Sanfilippo community the hope that a cure is on the horizon. The grant is not only a tribute to Dr. Fu's dedication to the field, but also to all the families and friends of Ben's Dream who have worked together for over a decade to fund this research."


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Contact: Erin Pope
Erin.Pope@NationwideChildrens.org
614-355-0495
Nationwide Children's Hospital
Source:Eurekalert

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