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Patient Stories
Harrison's story: How gene therapy could help against Duchenne muscular dystrophy
When Pam first heard about gene therapy, she thought it sounded like science fiction. She never would have guessed her son would be among the first patients to receive an innovative new gene therapy that's giving new hope against Duchenne Muscular Dystrophy (DMD).
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Our clinic is a certified Duchenne Care Center by the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy. We treat children of all ages, from diagnosis to their transition to adult care.
Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy and typically affects boys. It usually starts between ages 2 and 5 and affects many aspects of a child’s health, from their ability to walk to the strength of their heart and breathing muscles.
Children’s Health neuromuscular disease experts understand the specific ways that muscular dystrophy affects breathing, heart function and other aspects of health. We collaborate with specialists from pulmonology, cardiology, rehabilitation medicine, orthopedics, endocrinology and nutrition to give patients the best possible care.
We use preventive care to keep your child’s body strong and avoid complications. Our preventive approach includes devices that help support breathing to prevent respiratory illness, and heart medication that helps kids stay healthy and active.
We also prioritize normalizing everyday tasks and experiences like getting ready for school, playing with friends and getting around the community. Together, we are ready to help your child retain their strength, prepare for changes.