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.

Santi Beats the Odds: A Story of Perseverance

August 30, 2024 – Santi brings joy to everyone around him, despite his medical limitations. At only age 7, Santi has memorized the telephone numbers of all his favorite people, including some of his nurses at Nicklaus Children’s Hospital.

Tamryn Gets to Walk Again after Deep Brain Stimulation Treatment at Nicklaus Children's

July 15, 2024 – After Tamryn was diagnosed with TOR1-A Torsion Dystonia Type 1, a rare movement disorder that causes involuntary muscle contractions and abnormal movements, he underwent surgery at Nicklaus Children’s Hospital’s to have a Deep Brain Stimulator (DBS) implanted.

Sofia's Inspiring Journey with SMA

July 26, 2023 – Sofia was born with spinal muscular atrophy (SMA) type 2, a genetic condition that affects the spinal cord’s ability to control the muscles, decreasing and limiting mobility. In Sofia’s 6 years of life, she has already overcome more medical challenges than most adults.

Patient of the Month: Gianna

August 01, 2019 – After noticing that her daughter was unable to crawl, stand, or do the things her friend's children were doing at six months of age, Idalmis decided that it was time to seek help. Sure enough, after some consultations and genetic testing, Idalmis' gut feeling was right.

Patient of the Month: Astry

October 04, 2016 – After Astry was born, her parents noticed that she wasn't progressing as babies typically do. She wasn't able to lift her head and she showed a lot of muscle weakness. Her pediatrician recommended they take her to see a neurologist, and so Astry's parents brought her to Nicklaus Children's Hospital.