MDA’s first-ever combined Clinical & Scientific Conference kicked off today at the Hyatt Regency Orlando in Florida. MDA President & CEO Lynn O’Connor Vos and MDA Chairman of the Board Dr. R. Rodney Howell opened the event.
“MDA is proud to convene this esteemed group, and we are grateful that you have joined us for our first combined clinical and research conference,” Lynn said. “If we reflect for a moment on where we were as a community five years ago, we are making great progress. Disease-modifying therapies are no longer a thing of the future, and we are at the forefront of new treatment paradigms.”
Lynn recognized the more than 1,200 clinicians, researchers, and pharma partners there to attend two-and-half days of sessions highlighting the latest in neuromuscular disease research and care.
“We are all united by the urgent need to bring innovative care, cutting-edge clinical research and new breakthrough treatments to our community,” she said. “This conference will give all of you an opportunity to collaborate, engage, and share groundbreaking research and new approaches to clinical care.”
This year, April 15 was International Pompe Day, and Dr. Howell shared a personal story about meeting the family of Joannes Cassianus Pompe, who first characterized the symptoms of Pompe disease. His work led to a good idea of what Pompe looked like, Dr. Howell said, so that when the age of newborn screening began, babies with the disease could be identified and treated.
A conference session on Wednesday will take a closer look at the current state of newborn screening.
Also during the opening ceremony, in the tradition of MDA’s longstanding Clinical Conference, two White Coat Awards were presented to national sponsors of MDA. The awards are inspired by white coat rituals held in some medical schools when students transition from studying to caring for patients. This year, MDA presented an individual White Coat Award to Mansfield Energy owner and longtime MDA supporter Michael Mansfield, and a corporate partner White Coat Award to Casey’s General Stores.
Following was a keynote speech by the U.S. Food and Drug Administration’s Dr. Janet Woodcock, director, Center for Drug Evaluation and Research, and a panel of speakers discussing drug approval processes, pain points, and possibilities for improvement. Stay tuned to Strongly for in-depth coverage in the coming days.
MDA’s National Ambassadors rounded out the opening events by speaking briefly about their experiences as ambassadors and their lives in general.
Faith Fortenberry, 7, of Waco, Texas, lives with SMA. She’s been receiving Spinraza, the first FDA-approved disease-modifying treatment for SMA, since 2016. Today, Faith said at the conference, she can breathe better. She recently stopped breathing treatments and BiPAP use overnight, which her mom, Leeann Fortenberry, said has made life at school—“and everywhere”—so much easier.
“Breathing is everything,” Leeann said.
Justin Moy, 18, of Concord, Mass., told the conference attendees about his bioinformatics studies at college.
“As I’m sure a lot of you are aware,” he said, “biology and the sciences aren’t the easiest things in college but I’ve definitely had a lot of fun getting to learn what you all know and getting to see the two meld together. I want to be able to contribute to the fight against neuromuscular disease.”
Tana Zwart, 34, of Sioux Falls, S.D., is MDA’s newest National Ambassador. She said the support she’s had in her life has motivated her to work through challenges.
“I’ve always had a very tremendous support system in my friends and family and even my community at home,” she said. “They’ve always believed in everything I did and helped me get through things. My position definitely has always been ‘I can figure that out, I can make that work,’ and if there are things that I can do I’m ready to do it. Quitting is not really in my vocabulary.”