The 2017 Congenital Muscular Dystrophy (CMD) Scientific and Family Conference made for the largest-ever gathering of CMD-affected individuals, family members, and experts.
First-graders were racing wheelchairs down the hall. A semi-circle of young hockey players, all in power wheelchairs, could be seen catching each other up about what’s happened since their most recent tournament. Parents stayed to chat long after the sessions concluded because, even after a long day of learning and socializing, there’s always one more story to share.
These were the scenes this past July at the 2017 CMD Scientific and Family Conference (CMD SciFam), in Arlington, Virginia. It was the culminating event of a 5-conference series made possible through a Eugene Washington Engagement Award from the Patient-Centered Outcomes Research Institute (PCORI).
The meeting hosted 380 individuals; approximately 300 who had a CMD or who were the parent, spouse, or caregiver of a CMD-affected individual. The remaining 80 attendees were comprised of the world’s top experts in CMD clinical practice and research, including: investigators Dr. Carsten Bönnemann and Dr. Reghan Foley from the National Institutes of Health (NIH), Dr. Alan Beggs and Dr. Behzad Moghadaszadeh from the Beggs Lab and Boston Children’s Hospital, Dr. Ana Ferriero from the French National Institute of Health and Medical Research (INSERM), Dr. Felice Heller, pediatric cardiologist from Connecticut Children’s Hospital, plus pulmonary experts Dr. Oscar H. Mayer from Children’s Hospital of Philadelphia, Dr. Hemant Sawnani from Cincinnati Children’s Medical Center, and Dr. Robert Graham from Boston Children’s Hospital, and many others.
Additionally, a representative from the Food and Drug Administration (FDA) was present, and opportunities for specimen donation to the Coriell Institute’s CMD BioBank and the CMD Tissue Repository at the Medical College of Wisconsin were made available. Various vendors like VibraLung and PromptCare were there, too.
Sessions included subtype-specific care and research, as well as panel discussions, support groups, and CMD community member-led breakouts like the popular Vehicle Conversions & Modifications, led by Simon Cantos and Jennifer Gluck of Making for Special Needs
. Special Needs Financial Planner Chase Phillips held a financial planning session
that proved particularly helpful to the community.
Goals of the 3-day conference were four-fold:
- Educate and connect affected individuals and families with each other and with CMD experts
- Increase knowledge and collaboration among early-career and established scientists and researchers
- Raise public awareness about, and build advocacy around CMD
- Build momentum toward CMD-related clinical trials. Cure CMD is excited to share that through conference activities; the organization has already achieved these goals, and will continue to drive toward each objective in the coming months.
CMD SciFam was a success for attendees. One said, “It was nice to get our questions answered by experts, and to be in a room full of others like myself. Additionally, I also really enjoyed the discussion section. I don't get a ton of opportunities to talk to other disabled people my own age, let alone a room full of people with the same or very similar conditions, so that was a fantastic experience.”
The event was also vital to Cure CMD, though, as it helped hone the organization’s research strategy for the next 5 years. As the only U.S.-based patient-advocacy group that focuses solely on the 5 CMD subtypes, it’s critical that the organization have an ongoing dialogue with the community. The organization wants to know: What is most important to the individuals we serve? What would improve quality of life for them? How can we ensure they feel they have the resources they need to be educated and supported, and continue to advocate for themselves and the CMD community?
Fortunately, the team of experts present at CMD SciFam, along with Cure CMD staff, were ready to listen. One of the most exciting outcomes of the conference is the groups’s newly-developed road map for the future. One-, 3-, and 5-year plans for research in CMD will drive us toward our goals as we continue the pursuit of treatments and a cure. In the meantime, Cure CMD staff are available to answer questions and make connections via our website, Facebook home page, and multiple Facebook-based support groups.
“This 5 conference series, and in particular CMD SciFam, has reinvigorated the CMD research community after being able to interact directly with the people for whom they are working to find treatments,” said Cure CMD Director of Operations Rachel Alvarez. “Community members – new and seasoned – were reminded how critical their support and advocacy are to reach our goal. Our path has never been clearer, and we will continue the incredible momentum built over the last year to achieve our mission to find treatments and cures for congenital muscular dystrophy.”
Cure CMD’s 2016-2017 Cure CMD Conference Series
included 4 scientific meetings, each focused on a different CMD subtype (LMNA, COL6, LAMA2, and SEPN1), with a final culminating 2017 CMD Scientific and Family Conference, which invited affected individuals and their families/caregivers, scientists, clinicians, researchers, representatives from pharmaceutical companies, industry professionals, and medical journalists. Please visit www.curecmd.org
for more information, and check out the group’s SciFam slideshow here