Volume 33 | Number 3 | Fall 2009 COURAGE International Walking for Personal Sponsor a Child Let’s Give MPS Day a Decade, Health Records for a Cure a Standing MPS community and Still Make life easier New fundraising Ovation to… celebrates, honors Going Strong! and be an active program allows MPS individuals are and remembers MPS Society partner in your families to honored for their walk/run events healthcare participate in resilience, courage mark 10-year walk/runs by proxy and passion for life anniversary Do you have a personal story MISSION STATEMENT or an article idea for a future The National MPS Society exists to find 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 office (ground floor 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 reflect 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 ................................................................. 4 Development Director’s Letter .......................................................... 4 Affected Individual’s Name New Members ................................................................................. 5 Date of Birth Donations .....................................................................................6–7 Diagnosis Family News ...............................................................................8–10 International MPS Awareness Day ............................................11–20 Relationship Fundraising ...............................................................................21–29 Address Legislative Update ....................................................................30–33 City, State, ZIP Making Headlines .....................................................................34–35 Upcoming Events .......................................................................... 35 Telephone Remembering Our Children ......................................................36–37 E-mail Standing Ovation ......................................................................38–41 Family b $50.00 A Warm Welcome .......................................................................... 41 Foreign b $80.00 Research News: Hope for the Future ........................................42–48 Professionals b $75.00 Volunteer Spotlight ...................................................................49–50 Corporate Memberships Available Resources | Helpful Information ................................................51–59 Would you like your name to appear in our directory? b YES b NO MPS Classifications ....................................................................... 60 Board of Directors ......................................................................... 61 Would you like to receive Courage, the Society’s newsletter? b YES b NO Would you like our publications in b electronic (e-mailed) format or b 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 Sydney VanHook (MPS I), Rachel Cumpain (MPS IV) and Sasha Segal (MPS III) Correction: The article on page 15 in the summer issue of Courage by Eddie Bell (MPS I) was transcribed by his mother and caregiver Debra Bell. 1 “ Never doubt that a small group of thoughtful, Your Education/Publicity Committee has the committed citizens can change the world. immense undertaking of modernizing your Indeed, it’s the only thing that ever has.” Society’s Web site to not only make it responsive for families but more informative for the general This inspirational, yet pragmatic quote was public. We have had a number of federal written by Margaret Mead, an anthropologist legislators, foundations, and other non-profit who studied and worked on cultural and social organizations visit our Web site to authenticate issues. Although I have read a few periodicals on our Society. Our sister organizations rely on our Margaret Mead I always come back to this quote, Web site for up-to-date information. This is an and it remains very applicable today with our area which we must continue to keep updated Society. Without our devoted members, we would and supportive for our members and others. This have never grown so fast and helped so many committee is looking into other innovations that with MPS diseases. can help educate and bring about awareness to Although we all take great pride in the our cause. accomplishments made with our Society’s The Family Support Committee interacts with successes, we cannot sit on our laurels and expect our membership on a daily basis, and we are so the future to be bright. Remembering that many honored to be able to sponsor your child with an of our children do not have treatments and none educational scholarship, to financially assist your have cures, we must continue to push forth in loved one in obtaining hearing aids, to assist you many areas to ensure cures will be found and in attending our conferences and to learn your that MPS and related diseases will be eradicated ideas on how families need support from our President’s Letter President’s in our future. organization. The Family Support Committee Our goal is to continue to forge new continues to explore other services. paths and directions in a variety of Although we do not mention the Technical areas. As you recently read in our Support Committee as much as we probably previous Courage, a new path for should, these dedicated ad hoc committee our Society is to support and thank members take on technical aspects with pride, Dr. Emil Kakkis for his approach knowing that funding for researchers is vital to to “fixing” the FDA through his our mission of curing MPS and related diseases. foundation’s legislative efforts. Dr. They work closely with our Scientific Advisory Kakkis is primarily known as the chief Board on each and every grant the Society offers medical officer with BioMarin, and and awards. for introducing enzyme replacement therapy into our MPS community. Last, but certainly not least, our fundraising is With his retirement from BioMarin, another critical extension of our Society. For such Dr. Kakkis is not sitting home a small membership with these rare diseases, we swinging on his front porch, but produce some amazing efforts in so many areas: instead is trying to fix what he sees as bake sales, recitals, dances, golf tournaments a broken system when it pertains to clinical trials and our walk/run events. In the first six months for those with the ultra orphan diseases. Please of this year, we have already had more than 30 go to www.curetheprocess.org to read and sign fundraising events held by loyal members and up for Emil’s blogs. their families. These events raise many dollars and critical awareness in our local communities. Another area your Legislative Committee is Our families are not bashful in telling their pursuing is to have an MPS caucus to continue communities that “someone I love needs a cure.” to raise awareness on a federal level. We will Your board of directors is not bashful either. not stop there! We will continue on new paths, and combine efforts with other non-profit Although I give many kudos to those who actively organizations whenever we have common ground participate on all these committees, the praise for issues. This is evident with our combined efforts the successes really belongs to you, our members. with other nonprofits and fighting for H.R.1441, Instead of being cynical because of your diagnosis The Ryan Dant Healthcare Opportunity Act with MPS or related diseases, you take the time of 2009. to thoughtfully tell us when we are deficient in your expectations. You again give us your praises 2 continued for the successes our Society has accomplished to heels for Opal. Thank you for the calls, cards, Letter Executive Director’s help your loved one. Our board of directors and e-mails and wishes for Trent and Amy. staff acts upon all feedback we receive. As I end the President’s Message, I will leave you We have received many calls from enthusiastic with my late mother’s favorite quote by Winston families looking forward to our 2009 Disney Churchill which also is very applicable to the World conference in December. It will be