News and updates from the MDA community
Shining STAR: The MD STARnet project expands to learn more about neuromuscular disease
MD STARnet, a national data collection and research program, is building on its work in Duchenne (DMD) and Becker (BMD) muscular dystrophies and expanding to include other forms of muscular dystrophy. The program focuses on gathering information that will help determine if there are treatments that can change the course of a disease, including respiratory treatments and possible ways to treat and prevent cardiomyopathy.
The project, which is operated by the Centers for Disease Control and Prevention, has been gathering data on people with muscular dystrophy in six areas of the United States since 2006. It also is funded by the MD-CARE Act, which was updated and signed into law by the President in September 2014. The MDA community played a critical role in ensuring the passage of this game-changing legislation that provides the research and infrastructure needed to accelerate discovery and bring drugs to market for all nine forms of muscular dystrophy.
In those eight short years, MD STARnet has learned a lot about DMD and BMD and those who are affected by the diseases. In addition, the project has been responsible for recent articles about the benefits of corticosteroid treatments in respiratory and cardiac care, and for explaining that delays in diagnosis for boys with DMD are common.
As the program expands, it is hoped that researchers will find out similar information about other forms of neuromuscular diseases, as well as additional information from new research about social services, quality of life, and the challenges and successes of transitioning to adulthood with muscle disease, among other topics. Another focus of this expansion is increasing the number of sites where data is taken in order to reach more demographics.
In making its research decisions, the project’s leaders have reached out to organizations such as MDA to help them prioritize what they should be looking at.
“They’re engaging advocacy partners [like us] very closely,” says Jodi Wolff, MDA’s director of clinical services. “We’ve been an integral partner in helping them prioritize their research. A meeting was held in October with the MD STARnet research teams, CDC team and advocacy groups, where we worked together to identify research opportunities that could make the most significant impact on improving the quality of life for individuals with muscular dystrophy.”
Close-Up on ALS: ALS hits the big screen in two recent films
Two major motion pictures that premiered in November, the Stephen Hawking biopic “The Theory of Everything” and “You’re Not You,” starring Hilary Swank, brought amyotrophic lateral sclerosis (ALS) to the big screen by featuring main characters who live with the disease. Both films are generating Oscar buzz, particularly for the performances given by Eddie Redmayne as Stephen Hawking and Swank as Kate, a classical pianist who is diagnosed with ALS.
With the added attention from last summer’s Ice Bucket Challenge, ALS awareness is at an all-time high. MDA is leading the charge in the quest to find treatments and a cure for ALS.
Calling All Camp Volunteers: MDA summer camp is right around the corner!
MDA summer camp is always a blast for everyone involved. Attendees and volunteers alike spend a week in the outdoors having fun and building bonds that last a lifetime. If you’ve thought about volunteering in the past but couldn’t commit, there’s no better time to become a part of summer camp than now. Summer camp volunteers play a lot of different roles at camp, from counselor to helper to friend. It’s a great way to give back and have some fun at the same time. Volunteers must be able to lift and care for campers and assist with their needs. All prospective volunteers must complete an application and extensive screening process, including a criminal background check.
If you’re interested in becoming a summer camp volunteer, contact your local MDA office at 800-572-1717 for more details.
Back By Popular Demand: MDA announces Reagan Imhoff’s second year as MDA National Goodwill Ambassador
MDA Goodwill Ambassador Reagan Imhoff spent 2014 traveling across the country, raising awareness and promoting MDA’s quest to save and improve the lives of people with neuromuscular diseases. Her 2015 is shaping up to be similar, as MDA recently announced that Reagan will continue in her role as MDA National Goodwill Ambassador for another year.
“I’m excited to keep spreading awareness,” Reagan says.
Reagan and her family in 2014 visited places such as San Diego, Philadelphia and Daytona Beach to help people in cities across the country better understand how neuromuscular disease affects the MDA community. Along with her parents, Jenny and Joe, Reagan speaks to national partners and others about the urgent need to support the thousands of families like hers affected by neuromuscular disease. By attending various MDA events nationwide, Reagan has become quite the public speaker, charming her way into the hearts of everyone she meets.
While Reagan was quick to note that every place she went was “really great and awesome” and that she didn’t have one favorite, one particularly memorable stop was in Nashville, where she had a special experience with the musical group Rascal Flatts.
“I got to go up on stage with Rascal Flatts at the Grand Ole Opry,” she says. “We said ‘Hi’ to everyone and we told them about MDA. It was awesome. I colored with Rascal Flatts backstage.”
“Reagan is very aware of the challenges she faces as a person with spinal muscular atrophy (SMA), but she handles everything with courage and grace and never misses an opportunity to tell her story, speak about the support she receives from MDA and enlist someone in our fight,” says Jenny Imhoff.
As for Reagan’s plans as the 2015 MDA National Goodwill Ambassador, she hopes to continue to support MDA’s mission.
“I’m just really honored [to be Goodwill Ambassador], and I hope we can find treatments and cures,” she says.
Be sure to connect with Reagan on her Rally 4 MDA Facebook page.
Tomorrow’s Therapies: Design, Discover, Deliver: MDA’s third annual scientific conference to be held March 11–14, 2015, in Washington, D.C.
MDA is urgently committed to accelerating drug development and treatments for neuromuscular diseases. To achieve this goal, the organization hosts an annual conference series, with scientific and clinical conferences held in alternate years.
The 2015 MDA Scientific Conference will convene researchers and clinicians specializing in neuromuscular disease from around the world. The meeting will focus on genetics and genetic modifiers, identifying targets for therapies, new therapeutic approaches, mechanisms of disease, ways to measure progression of neuromuscular diseases and their response to treatment, and clinical trials. Time also has been set aside for late-breaking news.
Know Your Options
Though often overlooked, home- and community-based Medicaid waiver programs are important options for people with neuromuscular disease, especially those who require some type of in-home care. To qualify, people must have a disability that affects their mobility, learning or self-care. These programs are available in all states, and while their services vary from state to state, many cover services like personal assistance, in-home care and transportation.
To locate services in your area, visit eldercare.gov. Just type in your ZIP code and search for the local Area Agency on Aging.
It’s Shamrocks Season: Go green by buying shamrocks to help MDA conquer muscle disease
MDA Shamrocks — one of MDA’s largest fundraising programs — is starting again this February and running through St. Patrick’s Day. When shoppers go into select retail locations, such as Lowe’s, Kroger, Burger King, 7-Eleven and many more, they can buy and put their name on a paper shamrock, which hangs in the store during the fundraising event, to support MDA and the fight against muscle disease.
“The MDA Shamrock program is the largest St. Patrick’s Day fundraiser in the world,” notes Todd Hermon, MDA’s national vice president of retail partnerships. “Every single shamrock sold represents hope in the fight against muscle disease and for the families MDA serves. It also is a meaningful way for businesses and retail outlets to publicly show their support for our cause and our families.”
Stores and retail outlets are beginning to show their support not just in brick-and-mortar stores but in the digital world, as well. Customers making online purchases at participating stores now have the opportunity to contribute to MDA’s lifesaving mission. This is part of a larger effort on MDA’s part to reach out to customers in new ways. “More and more, we’re enhancing the program to go beyond just the paper shamrocks,” says Hermon. “Whether that’s digital or whether it’s additional awareness pieces at the gas pumps or pin pads, we want more customer engagement with supporting the families MDA serves so we can find treatments and cures.”
Shamrocks 2014: By the Numbers
MDA is always looking for local MDA families to get involved in their community efforts. If you’re interested in being a part of the program, email firstname.lastname@example.org.
Caring for the Long Haul: Select MDA clinics celebrate decades of service
MDA clinics, located in many of the nation’s top hospitals, provide comprehensive care, support and guidance through every step of the journey for people with muscle disease and their families. And some clinics have done just that every day for 50 or more years.
Remember to participate in MDA’s clinic survey at surveymonkey.com/s/mdaclinic. Each response helps us improve your MDA clinic experience.
In 2004, Gerald Brown of Denison, Texas, was looking for a wheelchair for his son, Joshua, who has limb-girdle muscular dystrophy (LGMD). He came across a website showcasing a motorized wheelchair that worked on rugged terrain. Unfortunately, it was just in prototype at the time, so Gerald couldn’t get one for his son.
Ten years later, Joshua, now a drummer in his high school’s marching band, needed a chair to help him march along with the rest of the band. Gerald remembered the rugged chair he had seen years ago and contacted the creator, Brad Soden. It turns out Soden, a veteran himself, had created the prototype for his wife who is a paraplegic and was now partnering with veterans groups to manufacture so-called “tankchairs” for wounded veterans. Soon, Joshua would have a tankchair of his own for riding through mud as well as a second adapted chair for marching with the band.
“I contacted Brad, and he jumped all over it,” says Gerald. “And since then he has been like a dear family member. He built Joshua a chair that travels 22 mph and has all the bells and whistles. He actually drives it with his foot so he can play the drums. It’s amazing.”
Joshua’s drumming chair — called a “speedster” — allowed him to experience the marching band in a new way.
“I had always played from the sidelines and watched from afar, seeing their movements, and I don’t even know how to explain it; it was really great to be out there doing the movements and doing the drills and not having to wonder what it would be like,” Joshua says.
While the speedster is great for marching, Joshua uses his tankchair for more recreational activities.
“The tankchair really allows me to be more mobile outdoors,” he says. “I can go in ditches and in the mud, places I couldn’t go with my normal chair. I go fishing, camping and all sorts of things like that.”
When Katie Dickey first told her internship adviser at Arizona State University that she wanted to travel to Uganda and help schools there to fulfill a requirement in her Global Studies program, the response wasn’t what she hoped for.
“[My adviser] said she didn’t think it was a good idea,” says Katie, who was born with distal spinal muscular atrophy (SMA) and now works as a seventh-grade teacher. “That was frustrating, and I left her office feeling really dejected.”
But Katie quickly rebounded and decided to figure out how to make the internship happen on her own.
“I went home for a few days and pouted and thought maybe I should do the internship in the U.S., but I recognized that’s the easy way out and these kids in developing countries don’t even have that option. So I talked to my parents about it and made a proposal for [my adviser] that she couldn’t say ‘no’ to.”
Her proposal was approved and soon she found herself in Uganda, working at a school for children with disabilities. While she found the work rewarding, it took an emotional toll on her.
“In Uganda, people with disabilities aren’t valued in any sort of way, and this was the only school in the country for people with disabilities,” she says. “It was tragic, and really an eye-opening experience.”
The trip also made it clear to Katie that the challenges people with disabilities face are similar no matter where you live.
“I met three boys that were quadriplegic or paraplegic [in wheelchairs], and I really related to them because sometimes the teachers treated them like they had mental disabilities and it just seemed like they were being held back from complete success because they were being treated differently,” she recalls. “That really stuck out in my mind.”
Katie says having this experience partly affected her decision to become a teacher and work in low-income neighborhoods through Teach for America.
“When I went to Uganda and saw the education system there, it made me think of the kids in the U.S. who don’t have access to education here, and, in my opinion, a lot of those kids are in the low-income community, so that’s what I’ve been doing for the past few years.”
While she loves her job, Katie isn’t sure what the future holds for her.
“I always thought my career path would have something to do with people with disabilities, but now that I’m teaching I realize that even though I’m not directly working with kids with disabilities, I’m able to educate 280 kids — and that people with disabilities can live productive lives and are valuable,” she says. “I’m not sure what I’m doing next year. I might continue teaching, and I’m also taking the LSAT. I would like to do international human rights law. One of my majors had an emphasis in human rights, and we never talked about people with disabilities.”