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Major News National MPS Awareness Day Power in Numbers Rare SUMMER 2013 courage Volume 37 | No. 2 03 05 28 35 43 Major News National Power in Rare Disease Upcoming Synageva, MPS Numbers Impact Events Ultragenyx Awareness Collaboration Report 2013 family and Lysogene Day between Mt. Sinai Survey uncovers conference to be all announce Families nationwide and Icahn School impact of rare held Oct. 24–26 in treatment progress find unique ways of Medicine offers diseases on San Antonio, TX. to honor those hope with new patient & medical Register online affected treatment communities now! 0081 courage v4_web.indd 1 6/12/13 5:28 PM The National MPS Society’s office (ground floor on left). Do you have a personal story or an article idea for a future issue of Courage? Please write to us and remember to send photos! ISSUE To submit information to Courage, please send text (preferably via e-mail) SPRING to the address below. Photos should be labeled whenever possible. Please SUBMISSION CUTOFF DATE note cutoff dates. Any information received after these dates will be January 1 included in the subsequent issue. ISSUE SUMMER The articles in this newsletter are for informational purposes only, and do SUBMISSION CUTOFF DATE not necessarily reflect the opinions of the National MPS Society and its April 1 board of directors. We do not endorse any of the medications, treatments or products reported in this newsletter, and strongly advise that you check ISSUE FALL any drugs or treatments mentioned with your physician. SUBMISSION CUTOFF DATE Courage reserves the right to edit content as necessary. July 1 ISSUE WINTER SUBMISSION CUTOFF DATE October 1 National MPS Society PO Box 14686 / Durham, NC 27709-4686 t: 877.MPS.1001 / p: 919.806.0101 / f: 919.806.2055 e-mail: [email protected] / web: www.mpssociety.org 0081 courage v4_web.indd 2 6/12/13 5:28 PM TABLE OF contents 01 02 Letter from the President ON THE COVER 03 Letter from the Executive Director 04 Letter from the Development Director Carolyn White (ML III) 04 Letter from the Program Director 05 National MPS Awareness Day 07 Family News Jerry Klemm (MPS I) 10 Fundraising News 14 Making Headlines 16 Standing Ovation Angelica Mendoza 19 Remembering Our Children (MPS IV) 20 Legislative Update 24 A Warm Welcome 26 Research News 37 Resources and Helpful Information 41 Donations 42 New Members 43 Upcoming Events 44 Classifications 45 Board of Directors MISSION STATEMENT 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. 0081 courage v4_web.indd 3 6/12/13 5:28 PM LETTER FROM THE 02 president EVERY YEAR IN THE FALL, I has funded more than $4 million can’t help but remember when my for research grants which have three children were diagnosed with helped bring about clinical trials MPS I. It has been 19 years since leading to treatments of many that week in October of 1994 when MPS disorders. The Society has I first learned about the world of evolved into an organization that rare genetic diseases—a world that not only supports research, it strives soon consumed my previous reality. to support the many needs and The information my wife and I challenges of its families through were given about MPS I was sketchy education, family assistance and and the outcome of the disorder, awareness. dismal. Thankfully, there have been The most significant change since many changes over the years that my family’s diagnosis has been years. As a result, the Society will have been a support and help to enzyme replacement therapy celebrate this achievement at our families receiving their own MPS or (ERT). When my children were 2013 Annual Family Conference in related disease diagnoses. diagnosed, we were told there San Antonio, Oct. 24–26, 2013, with The biggest change since that may not be a treatment available a Genzyme-sponsored luncheon. time has been the dissemination in their lifetime. We didn’t know While this is cause for celebration, of information, especially the about Dr. Emil Kakkis and Dr. Liz I realize it also is cause for sorrow— Internet. When we wanted to know Neufeld, two researchers desperate sorrow that 10 long years after more about MPS I in 1994, we got to find funding for their successful the first approval, many families in the car and drove to a library research project to treat an MPS I are still waiting. The only solace is with a medical section, or we made canine colony with ERT. that the future is bright. Research long-distance phone calls to the It wasn’t until the summer of is quickly progressing and is doctors we read about in books at 1997 that we learned of an ERT very encouraging. My prediction the library or in Courage. Fourteen clinical trial for MPS I patients. We is that the next 10 years will long months after my children submitted the necessary medical yield unparalleled progress in were diagnosed, we bought our first information for our two oldest discovering treatments for all of home computer. My wife and children and in April of 1998, the MPS and related diseases. I took turns on the computer, often our son, Spencer, was selected So, my hope is that a family reading information about MPS all as the fourth child treated with receiving an MPS diagnosis today night long. ERT. Within six weeks, we knew has a very different experience Another positive change has been it was helping him tremendously. than we did 19 years ago. While it the evolution of the National Our daughters participated in is still incredibly difficult and life MPS Society. When I made my the phase III part of the trial, and changing, many quickly discover first phone call to the Society, I five and a half years after the trial that a treatment is available, while learned there was little research began, the FDA approved the others find out about progress being funded—it was considered drug in the United States in 2003. toward a treatment for their sort of a black hole with no end in A few months later, the drug was syndrome. In all cases, they have sight. Money raised by the Society approved in Europe. easy access to a mountain of primarily went toward family This year marks the 10-year information about the disease and conferences and printing Courage. anniversary of approval of ERT for a Society that is able and willing Today, in addition to organizing MPS I. This approval paved a path to help. r family conferences and printing that allowed other MPS diseases to Courage, the National MPS Society receive approval in the following 0081 courage v4_web.indd 4 6/12/13 5:28 PM Steve Holland LETTER FROM THE executive director 03 FOR MOST OF THE WORLD, May 15 is just another day, but for everyone touched by MPS it is a day of recognition and awareness. This year it was much more than that. It was a remarkable day of announcements, announcements about treatments for our children. There has never been a time of such rapid growth in the world of Synageva MPS III B patient advisory board meeting MPS of natural history studies, new companies and clinical trials. It is MPS Society, at our conference last had the opportunity to talk with a testament to the significance of summer in Boston. Her research families from around the country. May 15 that we saw three press led to a publication in January in To put all this in perspective, it was releases that day. What an honor PLOSOne and a collaboration only 10 years ago when the first for our MPS community! announced May 15 between Mount treatment for MPS, Aldurazyme, Sinai School of Medicine and bene was approved, and we’ll have a Anthony Quinn, chief medical pharmaChem to conduct clinical special celebration at our San officer of Synageva BioPharm, studies of PPS in patients with MPS. Antonio conference. Hope to see noted in their press release, “We The Ryan Foundation will be are pleased to be able to support funding an initial study on MPS I you there! r the various organizations, patients and anticipates starting to enroll and families in their efforts to raise patients later this year. disease awareness around the world.” Laurie Turner and I joined Earlier in May, Lysogene representatives from Synageva announced receiving orphan drug in March when they hosted an status from the FDA. Lysogene has MPS III B advisory board meeting. conducted a successful safety gene Synageva is developing an enzyme therapy clinical trial in MPS III A in replacement therapy (ERT) for France and plans to expand their MPS III B and plans to enter into dialogue with the FDA so the Barbara Wedehase clinical trials in early 2014. United States can become part of their scope, allowing U.S. patients Ultragenyx announced on May 15 to benefit from future treatment. Jill Jepson, BioMarin; Stephanie Bozarth, National that their phase I/II clinical trial MPS Society vice president and MPS IV parent; Expect to hear announcements Barbara Wedehase, National MPS Society executive for MPS VII will begin this year in director; Michelle Samenfeld, BioMarin. the United Kingdom. Dr. Emil from Drs. Haiyan Fu and Douglas Kakkis, whose vision for Ultragenyx McCarty at Nationwide Children’s is treatments for rare diseases, Hospital in Columbus, OH. They developed the first ERT for an MPS are rapidly moving forward with disease, Aldurazyme for MPS I. You their gene therapy for MPS III A can read about Dr.
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