Elizabeth's Success Story


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Ka'lee is Running, Walking and Thriving After Gene Therapy for Muscular Dystrophy

June 19, 2026 – Ka'lee 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. Since his first treatment in 2024 he has achieved milestones that once felt out of reach.

Ignacio is Breathing and Living Stronger

March 13, 2026 – Ignacio was born with a rare congenital anomaly on his tracheal rings. He remained undiagnosed of his condition until he was evaluated for sleep apnea at 8 years old. The ENT specialists at Nicklaus Children's Hospital were able to diagnose the source of his breathing obstruction, snoring, and nasal congestion and performed a surgery to open his airways and widen his trachea.

Leading Pediatric Epilepsy Care for 40 Years

February 26, 2025 – For 40 years, Nicklaus Children's Hospital has been a leader in pediatric epilepsy. Learn more about the comprehensive care we offer for the most complex chronic cases.

Gene Therapy Offers New Hope for Children with Duchenne MD

January 23, 2025 – At Nicklaus Children's Hospital, gene therapy offers a promising treatment that dramatically improves life for children with Duchenne muscular dystrophy. Recognizing the potential wielded by a new gene therapy, Nicklaus Children’s became one of the first in the nation to offer ELEVIDYS-dystrophin soon after the FDA's 2023 approval.