Ka'lee, 9 years old, was diagnosed with Duchenne Muscular Dystrophy (DMD) at just two years old. One of four siblings, he is the only one in his family with this condition. His mother, Carmen, did not expect the diagnosis, and at first, didn't fully understand what it would mean for her son's future.
DMD is a rare genetic disease that affects approximately 1 in 3,500 boys. The condition is caused by the absence of dystrophin, a protein essential to muscle function. Without it, muscles progressively weaken, typically taking away a child's ability to walk during adolescence and leading to serious cardiac or respiratory complications by their 20s.
In 2024, Ka'lee received ELEVIDYS at Nicklaus Children's Hospital the first FDA-approved gene therapy for DMD. Developed by Sarepta Therapeutics, the therapy delivers a shortened, functional version of the dystrophin protein directly to muscle cells, targeting the disease at its genetic source. Nicklaus Children's was among the first hospitals in the nation to offer ELEVIDYS following its initial FDA approval in 2023.
Ka'lee's care was led by Dr. Migvis Monduy, a pediatric neurologist and Medical Director of the Neuromuscular and Movement Disorders Programs at Nicklaus Children's Hospital. "Ka'lee has shown improvement in mobility, and our goal is with the initial dosing and supporting medications, it will delay the progression of the disease. Our goal is for him to live a longer mobile and active childhood," said, Dr. Migvis Monduy, pediatric neurologist and Medical Director of the Neuromotor and Movement Disorders Programs.
Since his treatment, Ka'lee has been running, walking and engaging in physical activity for extended periods and has achieved milestones that once felt out of reach.
"Watching Ka'lee thrive today running, playing, keeping up with other kids is something I never dared to dream about. This therapy has given our family a future we didn't think was possible," said Carmen, Ka'lee's mother.