Monthly Archives: March 2012

Ultragenyx announces FDA and EMA granted orphan drug designation for UX003 for the treatment of MPS VII

On February 28, 2012, Ultragenyx Pharmaceutical Inc. announced that the FDA office of Orphan Products Development granted orphan drug designation for UX003 for the treatment of MPS VII. See the full press …

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“Understanding Clinical Studies Involving Enzyme Replacement Therapy” (video)

The video entitled, “Understanding Clinical Studies Involving Enzyme Replacement Therapy,” is now available www.biomarinclinicaltrials.com/MPSIVA.html. It was developed to help educate patients about ERT (enzyme replacement therapy) clinical studies, share patients’ …

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National MPS Society is now accepting Continuing Education Scholarship applications

Applications are now closed for this period.  We will be making announcements on the 2012 recipients on or before May 15, 2012. Please contact Laurie Turner, Program Director at laurie@mpssociety.org …

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Shire is now accepting applications for the ACES scholarship program

Now in its second year, the Shire ACES scholarship program is continuing last year’s success in recognizing academic achievement in the lysosomal storage disease (LSD) community. High-school graduates or GED …

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