Category Archives: News

News & Events

Society’s 2014 Research Grants Announced

The Application for Letter of Intent is now available here. The National MPS Society funds research that may lead to treatments for MPS diseases and solicits applications for innovative research …

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Information about new resources for families, specifically caregivers

The website at www.RareCaregivers.org is a guide for family caregivers of loved ones with rare diseases and addresses their distinct needs.  The resources available include: ·         Products, services, and technology …

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Don’t let Congress repeal incentives for rare disease drug development!

This year, it is likely that our members of Congress will pass tax reform legislation. One of the items that could be on the chopping block is a current tax …

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First Treatment for MPS IVA Approved

2013 MPS IV advisory committee hearing.

BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Vimizim™ (elosulfase alfa), the first specific therapy approved for the treatment of Morquio A (MPS IVA syndrome.)

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Annual EveryLife Art Contest

Calling All Rare Artists! The Annual EveryLife Art Contest has moved to Facebook to allow you to share your art with friends and to enable the community to vote and …

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BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome

FDA NEWS RELEASE FDA approves Vimizim to treat rare congenital enzyme disorder First drug to receive Rare Pediatric Disease Priority Review Voucher The U.S. Food and Drug Administration today approved …

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BioMarin Announces Selection of NAGLU, BMN 250 for the Treatment of Sanfilippo B

BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B (MPS IIIB) Please click here for the full BioMarin News Release

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Emory “All LSD” meeting on March 8, 2014

Click HERE for more information about the “All LSD” patient meeting about research being held on March 8, 2014 at the Emory Conference Center in Atlanta, GA. Patients, parents, and …

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13th International Symposium on MPS and Related Diseases

Click here to find out more about the 13th International Symposium on MPS and Related Diseases!

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MPS II Intrathecal Clinical Trial Update

MPS II Intrathecal Clinical Trial Update

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