When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...
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 moxeparvovec-rokl) ...
Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...
Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...
The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...
Khaleej Times on MSN
Fakeeh University Hospital sets new benchmarks in advanced gene therapy
Fakeeh University Hospital is emerging as a global destination for advanced gene therapy, delivering highly specialised ...
HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...
Imagine having a toddler who starts missing his developmental milestones. Then imagine sitting across the desk from a doctor who is about to share the results from a battery of tests ordered because ...
The FDA cited risk of IV infection and kidney toxicity in its letter. The company's CEO said the FDA issued no prior warning for the two concerns.
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