SPRING 2012 courage Volume 36 | No. 1 08 23 34 41 43 Spring Register Legislative Research Co-pay Cleaning Now! Advocacy Progress Assistance Progresses New board 2012 conference New MPS III B Shire, Genzyme members bring registration Society hires natural history offer programs to fresh skills, now open strategic firm to study, MPS VII offset expenses perspectives advance objectives ERT 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 TABLE OF contents 01 02 Letter from the President ON THE COVER 03 Letter from the Executive Director 04 Letter from the Development Director Jake Elston (MPS III) with siblings Hannah 04 Letter from the Program Director and Luke 05 Financial Summary 06 Board Minutes 08 New Board Members Joseph, Harly, Henry (MPS III), 10 Presidential Proclamation of Courage Melissa and 11 Fundraising News Haley Koker 20 Family News 23 Upcoming Events Kianna White 24 A Warm Welcome (MPS IV) 26 Holiday Photos 28 OSCAR Award 29 Remembering Our Children 30 Standing Ovation 32 Legislative Update 35 Making Headlines 37 Research News 43 Resources and Helpful Information 48 Donations 50 New Members 52 Classifications 53 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. LETTER FROM THE 02 president THE SOCIETy’S 2012 EMPHASIS on everything NEW was highlighted in our New Year’s card and the beautiful picture Jack Frye drew. His mother, Kim, stumped several of us to recognize exactly what the picture is. Yes, it’s a beautiful butterfly, but what else do you see? (See answer below.) Our Education/Publicity Committee is developing an International MPS Day art contest where we will choose our 2013 New Year’s card picture. We are benefiting by the expertise our NEW board members bring to the Society through their professions and passions. It’s helpful to not just have them asking “Why?” but also providing suggestions and alternatives. This is an effective tactic for growth and reNEWal, and we’re seeing early signs of that within all our committees. We are always on the lookout for NEW committee members. Contact me if you’re interested in serving on a committee. My children have been involved in several clinical trials and they, along with Amy and I, know the commitment involved for everyone. Clinical trials allow NEW treatments to be tested to determine their efficacy before being rolled out as the new standard of care for MPS. Being part of a clinical trial often means months of traveling to the trial site, not knowing if you are getting the treatment drug or a placebo, and whether it is working or not. All the while, the travel and treatment schedule disrupts school, home and family life. Now I am not complaining, because the opportunity to participate is truly a gift. However, the commitment of patients and their families participating in these trials must be acknowledged. With the NEW MPS IV clinical trial and the upcoming MPS VII clinical trial, more of you have made this commitment. I thank you for participating and paving the way to treatments In 2011 one of our sister MPS for those yet to be diagnosed. It’s very special to be a pioneer. organizations learned that a former treasurer of that organization Over the years the Society has partnered with several family foundations to removed almost $140,000 from offer research grants. For the inaugural year of our NEW Grand Challenge their accounts. Questions may arise MPS III Grant, Team Sanfilippo is partnering with us by providing financial such as, “Can that happen to us?” support. I hope to continue our collaborative grant funding with Team We want to reassure our members Sanfilippo and other family foundations to further increase the total that the National MPS Society investments in cures for our children. I encourage you to read the Scientific has internal controls in place that Advisory Board’s (SAB) Future Directions Committee’s grant funding provide oversight for our finances so recommendations on our Web site. I am grateful for the expertise of our SAB not just one officer or staff member as they help direct the Society’s grant program, so our donors can be assured has responsibility for our various their research dollars are invested wisely. accounts. Many of these controls are It’s not too early to register for the Boston family conference and the NEW required by our auditors and others events offered. Boston is a beautiful historic city, and we’re excited to be back have been initiated by the board. If in the Northeast. you have questions, please contact one of our board or staff members Have you figured out that picture yet? Well if you look closely, you will see for more information. that the outline of the butterfly’s wings are formed by two sweet MPS hands— those of Jack Frye (MPS II)! r Steve Holland LETTER FROM THE executive director 03 I’M NOT SURE I REMEMBER A NEW YEAR that began with so many NEW initiatives for the Society. All of us in the Society’s office are busy jumping from one project to another, many of them you’ll read about in this edition of Courage. Speaking of Courage, did you notice its fresh NEW look, color cover and color holiday photos? There’s more: The electronic edition of Courage is in full color! Even if you don’t receive an electronic edition, you can download it from the Members’ Only section of our Web site. I’d like to welcome our NEW board that Angela is a certified public Society will be offering a $235,000 members: Dawn Checrallah, Lisa accountant and served as treasurer, MPS III “Grand Challenge Grant” Todd and Roy Zeighami. You can therefore she is knowledgeable in 2012 with support from Team read about them, their families about our policies and database. Sanfilippo, to support an MPS III and their goals on page 8. Having attended our November board On the legislative front, we meeting, they eagerly began their submitted in January the committee work in January and nomination form for MPS I to the were active participants in our Health Resources and Services February board meeting. Stephanie Administration Secretary’s Advisory Bozarth began her tenure as vice Committee on Heritable Disorders president and is the NEW chair of in Newborns and Children. the Legislative Committee. Kim This is the first step—and the Whitecotton is the NEW chair of the first MPS disease nominated for Education/Publicity Committee. approval—to the list of newborn Thanks to a generous anonymous screening diseases. In addition, donation, the board approved M+R Strategic Services began clinical trial, or to study either a a NEW family support program, working with the Society’s legislative therapy or a biomarker that has the Emergency Relief Program advocacy program. The work of passed the proof of concept stage (see page 43). the Legislative Committee has but requires further pre-clinical become too extensive for our or clinical testing. This grant is in Bruce Makous, a professional volunteer committee, and rather addition to MPS II and MPS IV fundraiser and planned giving than hiring a new staff person, the grants. specialist, spoke at our February board elected to initially utilize a board meeting about planned consulting group. After talking with We have been working on the three giving—our latest fundraising our key stakeholders and receiving upcoming 2012 conferences— program. Mr. Makous discussed proposals from several consulting family, CYCLE and SPIRIT—that how we can take this program not groups, Ernie Dummann, Steve will be held in Boston in July. only to our membership, but to Holland and I talked with each of Registration materials have been our communities. the groups. We feel M+R is best mailed and are on our Web site. suited to move the work of the Society I encourage you to register early Angela Guajardo retired as board forward. You’ll be hearing from for the conferences and the hotel. member in December, then began them and the NEW programs we We are expecting this to be a big her NEW staff position in January as have planned throughout the year. conference. I hope to see you in part-time accountant. As the Society Boston! r has grown, the role of treasurer Thanks to a NEW proposal from has become more complex and our Scientific Advisory Committee’s time consuming.