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Volume 33 | Number 2 | Summer 2009 COURAGE Big Plans for Research Rediscover Your Focused and Legislative the Future News Resources Moving Forward Update National MPS BioMarin This issue packed Board of directors Obama Society announces announces new with helpful tackled wide array administration Continuing Education clinical trial information of issues in 2008 names new Scholarship winners for MPS IV A and unique leadership at opportunities FDA, removes stem cell research barriers Do you have a personal story MISSION STATEMENT or an article idea for a future The National MPS Society exists to fi nd cures issue of Courage? Please for MPS and related diseases. We provide hope write to us and remember and support for affected individuals and their to send photos! families through research, advocacy and awareness of these devastating diseases. The National MPS Society’s new offi ce (ground fl oor on left). Submission Cutoff Date Issue To submit information to Courage, please send text National MPS Society Jan. 1 ......................Spring (preferably via e-mail) to the address at right. Photos PO Box 14686 April 1 ...................Summer should be labeled whenever possible. Please note cutoff Durham, NC 27709-4686 July 1 ............................Fall dates. Any information received after these dates will t: 877.MPS.1001 Oct. 1 ...................... Winter be included in the subsequent issue. p: 919.806.0101 The articles in this newsletter are for informational f: 919.806.2055 purposes only, and do not necessarily refl ect the opinions E-mail: of the National MPS Society and its board of directors. [email protected] We do not endorse any of the medications, treatments or www.mpssociety.org products reported in this newsletter, and strongly advise that you check any drugs or treatments mentioned with your physician. Courage reserves the right to edit content as necessary. Table of Membership & CONTENTS Subscription Form President’s Letter .............................................................................2 Executive Director’s Letter ............................................................... 3 Name Program Director’s Letter .................................................................3 Development Director’s Letter ..........................................................4 Affected Individual’s Name New Members .................................................................................5 Date of Birth Donations .....................................................................................6–7 Diagnosis Upcoming Events ............................................................................ 7 2008 Recap .................................................................................8–9 Relationship Financial Summary ........................................................................ 10 Address Family News .............................................................................11–16 City, State, ZIP A Warm Welcome .....................................................................17–18 Standing Ovation ......................................................................19–22 Telephone Fundraising ...............................................................................23–25 E-mail Scholarship Recipients .............................................................26–27 Family b $50.00 Legislative Update ....................................................................28–31 Foreign b $80.00 Volunteer Spotlight ...................................................................32–33 Professionals b $75.00 Making Headlines .......................................................................... 34 Corporate Memberships Available Remembering Our Children ......................................................35–36 Would you like your name to appear in our directory? b YES b NO Research News: Hope for the Future ........................................37–42 Logo Order Form ......................................................................43–44 Would you like to receive Courage, the Society’s newsletter? b YES b NO Resources | Helpful Information ................................................45–55 Would you like our publications in MPS Classifi cations ....................................................................... 56 b electronic (e-mailed) format or b Board of Directors ......................................................................... 57 hardcopy (mailed) format Please send your membership form and check to: National MPS Society PO Box 14686 Pictured on the cover: Durham, NC 27709-4686 Nick Boyce, Kraig Klenke, Trey Lane 1 It is summer once again. During a time Kym Wigglesworth resigned. Austin and his wife, when all things become new and there Cheryl, are parents of Austin with MPS III and is a strong life force present all around reside in California. Austin was defeated in his us, your board has been diligently fi rst run for the board, yet immediately picked working on the new year’s tasks and up the phone and said he was willing and able to duties as always, but this year it seems volunteer his time to our Society. We thank the somewhat different. This year I have Noll family for their commitment. seen our committee chairs, committee You, as members, also have had a very busy members and our MPS staff working launch into summer by planning family more as a fi nely tuned team than ever fundraisers, MPS walk/runs, attending special before. There seems to be a renewed needs camps, and preparing and participating trust and your board is feeling very engaged. in MPS social gatherings. We are thrilled that Your working committees have taken on new you continue to submit your wonderful articles responsibilities and have rekindled all tasks. All for Courage. Sharing your stories, pictures, ideas chairs and committee members have put forth and networking helps all of us manage our lives many hours of devoted work and it shows in the with MPS. You, our members, continue to be products created. creative and educate others when it comes to For example, this December will be our seventh life with MPS. You have shown us several ways Disney conference, and for the second time in you enjoy celebrating National MPS Awareness a row we will be staying at a Disney resort with Day, from participating in local family and school President’s Letter President’s great prices and better access for our families. events to making contact with local and national We are proceeding with a makeover of our Web news media, and communicating with our site, new MPS fact sheets and booklet updates, congressional leaders to affect change for policies advocacy trips to Washington, DC, along with which will improve the quality of life for all. collaboration meetings with the National With all of this you still have time to devote to Institutes of Health and your Scientifi c Advisory your daily activities of caring for a loved one Board. Even though we are industriously working with MPS or reaching out to friends affected on speakers, childcare issues, etc. for Disney, work with MPS. I am not sure how you do it, but you is also underway on the 2010 family conference carry on every day with unrelenting energy and in California. We are reviewing research grants, zeal. The commitment and enthusiasm of our attending MPS international meetings and membership is overwhelming and that is why you partnering with other lysosomal storage disease achieve so much. You never give up. You never organizations. Enlightening others about National quit. You don’t just plop down on the sofa even MPS Awareness Day, creating our New Members if life is not going your way. You are willing to Spotlight, Standing Ovation Program, Continuing assist, support and extend a hand in friendship. Education Scholarships, along with our new U.S. We are all bound by a common bond and I am fl ag program for deceased children, all came thankful for it. As always, I am honored to serve about from ideas and suggestions from our this motivated membership. members. In February we welcomed Austin Noll to our board of directors to fi ll the spot open after 2 The importance of the National MPS Society’s Disease Network (LDN). We Letters Executive | Program Director’s research program was highlighted this year by met while attending the LDN the number of researchers submitting letters conference in San Diego and of intent, a total of 33. The Society has received have participated in conference global recognition as an organization that calls. Our letters of support are not continually supports quality research, and we only welcome, but required as part receive letters of intent from researchers around of the grant submission process. the world. During this period of reduced funding Collaboration among patient for this critical research, the money supplied groups, researchers and the NIH by the Society ensures that these innovative is crucial. research studies continue. Our research money Plans for the 2010 International MPS Symposium also fi nancially supports young researchers, in in Australia are under way, and I have been asked hopes MPS research will be a lifelong pursuit. to serve on the program committee. Leaders Our Scientifi c Advisory Board Research Review from many international MPS organizations Committee will read the 12 proposals selected will attend the Disney conference. The National for fi nal consideration of funding for the fi ve MPS Society is hosting the yearly meeting of the grants the Society is offering this year.

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