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Tiny cell “antennas” may help explain why some babies are born with heart defects
A newly discovered communication system in the microscopic “antennae” of cells may help explain how some congenital heart defects develop. When genetic mutations disrupt this system, the effects may extend beyond the heart to organs including the brain, kidneys, and skeleton.
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20 students with DMD are Sarepta scholarship winners
Twenty college students with Duchenne muscular dystrophy (DMD) will receive awards of up to $5,000 through Sarepta Therapeutics‘ Route 79 program. “As we mark the ninth year of this program, we continue to be inspired by the commitment these students show to their education and their futures,” Diane Berry, PhD, Sarepta’s executive vice president and…
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DJing with DMD shows how music connects us beyond disability
In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full…
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Life with Lily: Preparing for College with a Neuromuscular Disease
As I prepared for college after graduating high school, my experience mirrored that of many young adults in several ways. Like my peers, I searched for a welcoming campus culture, a strong program in my intended major, and a university that offers meaningful opportunities for collaboration, research, and learning. However, my journey differed fundamentally because…
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FDA Approves First Muscle-Targeted Therapy for SMA: A Conversation with Dr. Se-Jin Lee
The FDA has approved Isembyld (apitegromab-mstn), the first therapy designed to target muscle health and regeneration in people living with spinal muscular atrophy (SMA). This milestone adds to the progress made by genetic therapies for SMA and provides families with a new option focused on improving muscle strength and daily function. Se-Jin Lee, M.D., Ph.D.…
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Life is so busy that a week of medical appointments sounds relaxing
My life is always in motion, and we are in a busy season as my family prepares to move into a new house, one that is being modified for accessibility. I share my life with my husband, Jason, and our seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and…
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Overcoming denial to build a full life with Becker MD
In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full…
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Phase 3 trial of experimental DM1 treatment fails to hit main goal
A Phase 3 clinical trial testing delpacibart etedesiran (del-desiran), an experimental treatment for myotonic dystrophy type 1 (DM1), failed to hit its main goal. The Phase 3 HARBOR trial (NCT06411288) enrolled nearly 160 people with DM1, ages 16 to 65. Participants received seven infusions of either del-desiran or a placebo over the course of about…
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My FSHD journey revealed new strength I never knew I had
In recognition of Muscular Dystrophy Awareness Month in September, the Muscular Dystrophy Awareness Month campaign features a series of stories highlighting the real-life experiences of people affected by muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, or X for more stories like this, using the hashtag #MDAwarenessMonth, or read the full…
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In Case You Missed It…
Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
