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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First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kidsThe U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy. The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death. The approval came despite a long list of concerns from some FDA scientists about the company's research. But patients, physicians and and parents urged approval at a public meeting in April. The drug received accelerated approval, meaning the FDA has the option to revoke its use if further studies don't confirm it works.
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First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kidsThe U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy.
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