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) ...
Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
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Sarepta posts encouraging three-year data for Duchenne gene therapy
Sarepta Therapeutics Inc. (NASDAQ:SRPT) released three-year follow-up results from its EMBARK study, showing that its gene ...
Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...
EPIDYS trial met primary endpoint demonstrating statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date Median follow-up of over 3 additional years ...
Duchenne muscular dystrophy (DMD) is an inherited muscle disorder that causes progressive breakdown of muscle tissue. Symptoms typically begin in early childhood, and most parents and caregivers ...
Sarepta Therapeutics demanded a prominent patient advocacy organization censor a video that contained pointed criticism of the company’s recently approved gene therapy for Duchenne muscular dystrophy, ...
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