National MPS Society Celebrates International MPS Awareness Day on May 15, 2008 Society partners with biopharmaceutical companies to host events at Children's Hospital Oakland Research Institute in Oakland, CA, and at North Carolina General Assembly Legislative Building in Raleigh, NC for families and medical professionals to celebrate and remember MPS patients. For More Information on MPS Awareness Day 2008, please contact: Barbara Wedehase Executive Director, National MPS Society (919) 806-0101 barbara@mpssociety.org Jasmine Chng Waggener Edstrom Worldwide (617) 576-5877 jchng@waggeneredstrom.com NEWS FACTS May 15, 2008 is International MPS Awareness Day 2008. This is a day for families and medical professionals to celebrate and remember people with mucopolysaccharidoses (MPS) diseases, a family of rare, potentially fatal genetic diseases that occur because of the inability to produce specific enzymes. The National MPS Society will host celebration events at the Children's Hospital Oakland Research Institute in Oakland, CA and North Carolina General Assembly Legislative Building in Raleigh, NC. Patients, families and physicians are welcome to join in the celebrations. The National MPS Society is partnering with biopharmaceutical companies BioMarin, Genzyme and Shire Human Genetic Therapies to commemorate this extraordinary day and educate the general public and medical community about the importance of early detection and treatment. EVENT INFORMATION Children's Hospital Oakland Research Institute (CHORI), Oakland, CA Time: 10:30 a.m. – 12:30 p.m. PDT Location: Children's Hospital Oakland Research Institute (CHORI) Library 5700 Martin Luther King Jr. Way Oakland, CA 94609 Local contact: Jasmine Chng jchng@waggeneredstrom.com North Carolina General Assembly Legislative Building, Raleigh, NC Time: 10 a.m. – 12 p.m. EDT Halifax Mall (located just outside the building) 16 West Jones Street Raleigh, NC 27601 Local contact: Amy Petty amyp@waggeneredstrom.com QUOTES • "The National MPS Society advocates for our children and their families. We do this by funding research programs to help find cures for MPS and related diseases, give families a better quality of life and increase awareness about MPS diseases within the medical community with programs such as 'Join The Search'," said Barbara Wedehase, executive director of the National MPS Society. "As we're scattered throughout the country, we've created International MPS Awareness Day as our very own day to help establish a sense of community for us to celebrate together." • “I have been caring for individuals with mucopolysaccharidosis (MPS) for greater than 20 years, and it’s rewarding to see the progress we’ve made in managing and treating these rare diseases,” said Joseph Muenzer, M.D., Ph.D., Professor of Pediatrics, University of North Carolina at Chapel Hill and a key investigator in the successful enzyme replacement clinical trials for MPS I and MPS II. “We now have treatments for three out of the seven MPS diseases, but there is still much work to be done as we need to develop better screening methods to allow early detection and to develop treatments for all individuals with a MPS disease.” • "Children's Hospital & Research Center Oakland has been working closely with both the National MPS Society and Dr. Muenzer and led the clinical trials for the first enzyme replacement therapy treatment for MPS VI," said Dr. Paul Harmatz, M.D., pediatric gastroenterologist at Children's Hospital Oakland. "We are thrilled with results to date and look forward to the opportunity for all patients with MPS VI to benefit from this important therapy." BACKGROUND INFORMATION ON MPS • Mucopolysaccharidoses (MPS) and related diseases are genetic lysosomal storage diseases (LSD) caused by the body's inability to produce specific enzymes. • There are seven MPS diseases, MPS I, MPS II, MPS III, MPS IV, MPS VI, MPS VII, and MPS IX. • Symptoms of the diseases may vary from one syndrome to another although there are many similarities. Affected individuals often have mental retardation, cloudy corneas, short stature, stiff joints, speech and hearing impairment, chronic runny nose, hernia, heart disease, hyperactivity, depression, pain and a dramatically shortened life span. • There is no cure, but treatments such as recombinant DNA derived enzyme replacement therapy are available to treat three of the MPS diseases— MPS I (Hurler, or Hurler-Scheie syndrome), MPS II (Hunter syndrome) and MPS VI (Maroteaux-Lamy syndrome)and hematopoietic stem cell transplantation for MPS I and MPS VI. However, it is devastating to know that there are still many families around the country who are unaware that treatment, and in turn, hope, exists. • For more information, please visit the Q&A section of the site. PHOTOS Joseph Muenzer, M.D., Ph.D. University of North Carolina at Chapel Hill Paul Harmatz, M.D., Children's Hospital & Research Center Oakland GRAPHICS/LOGOS ONLINE PHOTO ALBUMS • Events Photo Album • Faces of MPS RELATED LINKS • Join The Search • MPSVI.com • Naglazyme Family Site (MPS VI) • MPS 1Dieasease.com • Aldurazyme Family Site (MPS I) • Hunterpatients.com (MPS II) • Elaprase.com (MPS II) BOILERPLATE STATEMENTS The National MPS Society exists to find cures for MPS and related diseases. We provide hope and support for affected individuals and their families through research, advocacy and awareness of these devastating diseases. Since 1974 the Society has supported individuals and families affected with MPS and related diseases. We are governed by a member-elected volunteer Board of Directors, many of whom are parents of children with MPS. We benefit from the expertise of a Scientific Advisory Board, comprised of world-class physicians, researchers and medical professionals throughout the world.