Connecticut Children’s made medical history this week as it became the first health system in the state to administer gene therapy for Duchenne Muscular Dystrophy (DMD). The groundbreaking procedure will be conducted by renowned pediatric neurologist, Dr. Gyula Acsadi.
Duchenne Muscular Dystrophy is a rare genetic disorder that affects approximately 1 in 3,500 male births. It leads to progressive muscle degeneration and weakness, loss of movement and heart failure, significantly impacting the quality of life for those diagnosed. Connecticut Children’s commitment to advancing medical care and embracing innovative treatments marks a significant step forward in the fight against this devastating disease.
Dr. Gyula Acsadi, Chief of Pediatric Neurology and Director of the Muscular Dystrophy Association (MDA) Care Center and Neuromuscular Program at Connecticut Children’s, expressed his enthusiasm for this groundbreaking achievement, stating, "Administering gene therapy for Duchenne Muscular Dystrophy represents a significant milestone in pediatric neuromuscular diseases. Connecticut Children’s has been using gene therapy treatment for spinal muscular atrophy, successfully. We are excited to offer hope and transformative treatment to patients who face the challenges of this debilitating condition."
Connecticut Children’s President and CEO, Jim Shmerling, emphasized the health system’s dedication to providing cutting-edge care for pediatric patients. "This historic moment reflects our commitment to pushing the boundaries of pediatric healthcare. Being at the forefront of Duchenne Muscular Dystrophy gene therapy underscores our relentless pursuit of innovative treatments to enhance the lives of children."
The gene therapy approach aims to address the root cause of Duchenne Muscular Dystrophy by introducing a functional microdystrophin gene into the patient's muscle cells. Dystrophin is a protein crucial for muscle function, and its deficiency in DMD patients leads to progressive muscle degeneration.
For the child who will be receiving this groundbreaking treatment, the potential impact is life-changing. Gene therapy offers the prospect of slowing the progression of the disease, providing newfound hope for improved mobility and quality of life.
Connecticut Children’s will continue to monitor and support the patient throughout their journey in the comprehensive neuromuscular clinic, contributing valuable insights to the ongoing research and development of gene therapies for Duchenne Muscular Dystrophy.
As the first health system in Connecticut to embark on this groundbreaking path, Connecticut Children’s reinforces its commitment to advancing pediatric healthcare and bringing hope to families facing the challenges of rare and complex conditions. It’s innovation and care that is truly beyond imagination.
For media inquiries, please contact: Monica Buchanan, Sr Dir Enterprise Communications, 352.219.0860, mbuchanan [at] connecticutchildrens.org.
About Connecticut Children’s
Connecticut Children’s is the only health system in Connecticut 100% dedicated to kids. That means high-quality care across more than 30 pediatric specialties in 40+ locations throughout Connecticut, Eastern New York and Western Massachusetts. One team on a mission to improve access to healthcare from before birth and into adulthood. One team pushing the limits with breakthrough research and community partnerships so we can help you focus on what matters most: your family’s wellness journey. When you choose Connecticut Children’s, you can expect care Beyond Imagination.