Give to the 2014 Annual Fund: Chair Lynn Hopkins
“I hope the world is kind to Michelle because Michelle is the kind of spirit that makes the world a better place.” The National MPS Society is making significant gains …
28th Annual Family Conference – Disney
The Family Conference in Disney is from December 18-20. Click here for the link to register! Registration deadline – November 26, 2014.
40 Years of Achievements Gala photos!
The photos from our 40 Years of Achievements Gala are now available! Click here to find out where to view them!
First Treatment for MPS IVA Approved
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.)
Mucopolysaccharidoses (MPS) and related diseases are genetic lysosomal storage diseases (LSD) caused by the body's inability to produce specific enzymes. Normally, the body uses enzymes to break down and recycle materials in cells. In individuals with MPS and related diseases, the missing or insufficient enzyme prevents the proper recycling process, resulting in the storage of materials in virtually every cell of the body.