🔬 After decades of research, the FDA has approved the first-ever gene therapy for Duchesne muscular dystrophy, developed at Nationwide Children's Hospital.
💉 Dr. Jerry Mendel, who launched the gene therapy research project in 1999, calls the newly approved therapy the biggest event in the history of muscular dystrophy.
👦 The gene therapy offers hope for restoring movement and motion in children with Duchesne muscular dystrophy, improving their quality of life.
🏥 Ohio medical team makes breakthrough in muscular dystrophy treatment.
🧬 Muscular dystrophy is a genetic disorder that progressively weakens muscles.
💡 Dr. Men's treatment involves using a virus as a delivery system.
💡 An Ohio medical team has made a breakthrough in treating muscular dystrophy using viral genes injected into the veins of children to replace disease genes and restore muscle function.
🙌 The FDA has approved this treatment, leading to emotional reactions from the medical team and positive impact on young lives.
💪 Dr. Mandel and nurse practitioner Kelly Layman collaborate to implement the treatment, with Layman taking care of practical tasks and Dr. Mandel taking on the role of the 'good guy'.
🏥 Ohio medical team achieves breakthrough in muscular dystrophy treatment.
💝 Patients express gratitude to Dr. Mendel for the successful gene therapy treatment.
🔜 Serepta Therapeutics plans to make the treatment widely accessible in the near future.
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