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FDA asks Sarepta to stop shipping gene therapy
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Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...
Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...
Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days ...
The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene ...
New gene correction therapy for Duchenne muscular dystrophy. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2020 / 01 / 200127134851.htm ...
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...
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