Photo courtesy of Meredith Huml Day 28 of 30 This is Meredith Huml’s story: Independence has been a goal of mine for many years. I haven’t always chased after it in the healthiest manner. I need help in different ways than most people. I don’t like to make unusual…
Treatment with the investigational cell therapy CAP-1002Â significantly improved arm and heart function in boys and young men at advanced stages of Duchenne muscular dystrophy (DMD), according to final data from the HOPE-2 clinical trial. The Phase 2 trial also “met various skeletal and cardiac endpoints [goals] suggesting clinically…
Photo courtesy of Scott Bisi Day 27 of 30 This is Scott Bisi’s (@cougarhousegarage) story: My name is Scott Bisi, and I have always had big dreams. Living with muscular dystrophy (MD) and wanting to follow my dream of working in the automotive industry was challenging at first.
Photo courtesy of Mindy Henderson Day 26 of 30 This is Mindy Henderson’s (@mindyhendersonspeaks) story: When my daughter was 12, I went with her to a parent’s night at a community event. We got into small groups and the kids each had to say what they liked best…
Photo courtesy of Hannah Lowe Day 25 of 30 This is Elizabeth Lowe’s story: My grandson Austin was diagnosed with LMNA-related congenital muscular dystrophy (L-CMD) just before the pandemic lockdown in early 2020. My daughter, Hannah, and her husband, Mark, had noticed that Austin had stopped picking up…
Photo courtesy of Faye Flatt Day 24 of 30 This is Faye Flatt’s story: I was diagnosed with Facioscapulohumeral muscular dystrophy (FSHD) at age 21 when my cousin was referred to a neuromuscular specialist who asked to see family members with similar physical characteristics. My mother was diagnosed…
I love the everyday, ordinary moments of motherhood. Maybe I’m crazy, but the laundry (no matter how never-ending), groceries, menu planning, and chauffeuring of kids all bring me so much joy! Duchenne muscular dystrophy can be a thief of that joy. Duchenne is hard to plan for, and because…
In August, my 2-year-old son, Alfie, became a big brother. While bringing a new baby into the world is an important life event for any family, when you are already raising a child with LAMA2-related muscular dystrophy, there can be even more to contend with. For about six…
Photo courtesy of Anjélica RamÃrez Day 23 of 30 This is Anjélica RamÃrez’s story: My name is Angélica RamÃrez. I just turned 23 years old. I am a writer and a student in my penultimate year of medicine. Throughout my life, I have suffered great adversities associated with my…
Translarna (ataluren) treatment delayed the loss of walking abilities by more than five years in boys with Duchenne muscular dystrophy (DMD) who carry nonsense mutations in their DMD gene, according to a study based on real-world data. The investigational therapy also slowed lung function decline by almost two…
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