The FDA has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children 4-5 years of age. Get the details on how Elevidys could help pediatric patients.
"The FDA remains committed to facilitating the development of innovative new therapies to reduce the impact of debilitating diseases and to improve outcomes and quality of life for those affected," he added.DMD is a rare muscle disorder that affects around one in every 3,500 male births globally, according to theThe FDA has approved the first gene therapy for the treatment of Duchenne muscular dystrophy in children ages 4 through 5 years of age.
As the disease progresses, most patients will require a wheelchair by the time they reach adolescence.The symptoms may include trouble walking and running, fatigue and learning difficulties.and trouble breathing. The mutation prevents the creation of this protein, ultimately causing muscle weakness and mobility issues.Current treatments for DMD aim to improve symptoms on an individual basis — but they do not address the genetic cause.
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