2015 Annual SMA Conference Info 4 Family Support 8 Clinical Trial Updates 24 Legislative Updates 54 Fundraising and Chapter Updates 70 WELCOME
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SPRING 2015 DIRECTIONS 2015 Annual SMA Conference Info 4 Family Support 8 Clinical Trial Updates 24 Legislative Updates 54 Fundraising and Chapter Updates 70 WELCOME VISION AND MISSION STATEMENT Cure SMA leads the way to a world without spinal muscular atrophy, the number one genetic killer of infants. We fund and direct comprehensive research that drives breakthroughs in treatment and care, and we provide families the support they need for today. OUR VALUES Innovation We are a non-profit, 501(c)3 tax exempt organization. Funds will be specifically Our commitment to a treatment and cure is not just about seeking directed to scientific, educational or solutions—it’s also about creating them. We’re working with some literary purposes in keeping with a of today’s sharpest minds to advance a diversity of approaches and charitable organization. The organization champion the most promising discoveries and methods. is proud to provide funding to the Cure Balance SMA newsletter and website, which provide information and networking As relentlessly as we pursue a treatment and cure, we are also opportunities to its members. strategic. We know the fastest way to a future without SMA is to take a comprehensive, unbiased approach to research and maintain a balance Submissions of optimism and realism. To submit articles or make other Collaboration contributions to our newsletter, please Our community is everything to us. We would not have made it this contact us at: [email protected]. far in our fight without the invaluable contributions of our researchers, Digital images are encouraged! doctors, and families. Together, we are—and always will be—stronger Send your digital pictures to: than SMA. [email protected]. Respect Change of address There is no “right way” to live with a disease like spinal muscular atrophy. Every person’s experience is different, and it’s every family’s Send changes, including ZIP code to: right to decide what SMA means for them. [email protected] or call 800.886.1762 or mail to: Cure SMA Membership, 925 Compassion Busse Road, Elk Grove Village, IL 60007 Thanks to the Cure SMA community, no person is ever alone in facing ©2015 Cure SMA. All rights reserved. this disease. We offer unconditional support to people affected by SMA No portions of this publication can be and communicate openly and honestly, giving them clear and accurate reprinted without written consent from information. Cure SMA. Determination Cure SMA does not support or endorse Our work is not done until we have a treatment and cure, and we’ll any particular treatment or therapy. remain strong in our fight no matter what challenges come our way. Information contained in this newsletter should not be used as a substitute for consultation with a qualified healthcare professional. On the Cover: Evan Vaudry, SMA Type II DIRECTIONS Spring 2015 2 CURE SMA Erin Trainor Memorial Fund Reaches Milestone Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person’s ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. Since 1984, we’ve directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children’s bodies, extend life, and lead to a cure. In February 1994, Thanks to the dedication of the Trainors, Barbara and Gene and the generosity of our donors, they We have deep expertise in every aspect Trainor lost their recently surpassed the 2/3rd mark on of SMA—from the day-to-day realities five-month-old the way to their goal, with over $667,000 to the nuances of care options—and daughter Erin to raised. until we have a cure, we’ll do everything SMA type I. Throughout the past 20 we can to support children and families years, the Trainor family, friends, and The Erin Trainor Memorial Fund exists affected by the disease. colleagues have raised funds for Cure to provide scholarships for newly diagnosed SMA families to attend Learn more about how you can help SMA through annual events such as The Annual SMA Conference. Not us reach a treatment and cure at www. the Chesapeake Crab Feast and the only will the ETMF allow families the cureSMA.org. Chesapeake Charity Golf Classic. In addition, they have provided comfort opportunity to participate in this three- to numerous families by forming the day event, but it will also provide each Chesapeake Chapter in Baltimore, with the opportunity to gather critical Maryland. care and daily living information from experienced health care professionals This led them to establish the Erin as well as other families living with a Trainor Memorial Fund, to honor their similar diagnosis. daughter’s memory by bringing newly diagnosed families together during “We truly see Cure SMA as a gift of the Annual SMA Conference and hope. This gift has blossomed over the strengthening the SMA community. The years through supporters like you,” say Trainors have set an ambitious goal: to Gene and Barb Trainor. raise $1 million through the Erin Trainor Memorial Fund. DIRECTIONS 3 Spring 2015 2015 CONFERENCE 2015 Annual SMA Conference JUNE 18TH– JUNE 21ST, 2015 KANSAS CITY, MO, AT THE WESTIN CROWN CENTER Every year, Cure SMA sponsors a conference to bring together the leading Known as the City of Fountains and SMA researchers, clinicians, and families living with SMA. Cure SMA has home to great jazz music and barbeque, been hosting the Annual SMA Conference since 1989. The weekend is filled Kansas City is an urban town with with a wide variety of workshops, keynote sessions with leading researchers, Midwestern charm, there are many a family- friendly poster session, a memorable children’s program, a family things to do for all ages. The Westin fun fest and carnival, teen and adult social activities, a dance party, and many Kansas City at Crown Center is in the opportunities to connect and interact with families and receive first-hand heart of it all. The Crown Center’s updates from the researchers. various shops, restaurants and theatres encircle the hotel. Some of the many Every year we look forward to reuniting as a community at this conference attractions include: The Science City and showing our support for others. As always, the family conference at Union Station, The LEGOLAND® and the researcher meeting run alongside each other. This is the largest Discovery Center, SEA LIFE Aquarium, conference in the world for those affected by SMA, and also for those Kemper Museum of Contemporary Art, involved in providing support and care for SMA patients. There is no other The Power and Light District and many program like it. The interactions between the researchers and families at this more. Come on along and enjoy the sites one conference are extremely special. The annual conference also provides and sounds of Kansas City. the children an opportunity to make new friends and have a great time. We are expecting another great turnout of over 1,100 attendees. The Westin Kansas City at Crown Center has been carefully chosen as the 2015 destination for the Annual SMA Conference. The hotel is encompassed by Hallmark’s Crown Center, which offers 85 acres of shops, restaurants, and attractions. Each of the 724 guest rooms is equipped with a mini-fridge, coffee maker and 37” flat panel TV along with other modern amenities. The hotel offers a variety of restaurants along with 24-hour room service. For your convenience there is complimentary wireless Internet in the lobby and public areas along with premium internet available in the guest rooms. Bring your suit along to enjoy the outdoor heated swimming pool. To register for the Annual SMA Conference, please visit www.cureSMA.org. You must first register for the conference before you can reserve your discounted hotel room rate of $135 per night. DIRECTIONS Spring 2015 4 2015 CONFERENCE NEWLY DIAGNOSED CONFERENCE PROGRAM Thanks to the generous funding provided by the Erin Trainor Memorial Fund, Cure SMA covers the registration fees for the Annual SMA Conference for all families newly diagnosed since the last conference. The mission of the Erin Trainor Memorial Fund is to generate substantial funds to be able to provide conference scholarships, allowing newly diagnosed SMA families/individuals the opportunity to attend the Annual SMA Conference. Our goal is to allow as many newly diagnosed SMA families as possible to attend and experience the benefits of this amazing conference. The meeting gives families the opportunity to gather critical care and daily living information early after diagnosis, to learn directly from experienced SMA physicians, and to network with other families. This program is automatically offered to all newly diagnosed families that SMA CONFERENCE GOALS contact Cure SMA. 1) To allow networking between For more information, please email [email protected] researchers, medical providers, EXHIBITOR OPPORTUNITIES patients, and families 2) To educate researchers on the latest The Annual SMA Conference is a wonderful opportunity to promote your research advancements company or products to SMA families, medical professionals, and researchers 3) To attract the best researchers from all over the world. By hosting an exhibitor table or booth, you will be able to the SMA field and encourage to personally meet with many families and medical professionals, as well as be collaborations prominently displayed throughout the conference and online. 4) To educate medical providers, patients and families on the basics For more information, please email [email protected] of SMA SPONSORSHIP OPPORTUNITIES 5) To update medical providers, patients, and families on the latest The Annual SMA Conference does not happen without a tremendous amount research and medical progress of work and support.