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Muscular dystrophy breakthrough: FDA approves first-ever gene therapy for rare children’s disease

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age.

Pediatric patients who have a confirmed mutation in the DMD gene and do not have any preemptive medical conditions will have access to the drug, called Elevidys.

“The approval will permit the treatment of many boys throughout the U.S., and this will then extend to Canada and the European Union,” Dr. Jerry Mendell, co-inventor of the gene therapy, told Fox News Digital. “This means that treatment will extend to virtually hundreds of boys and significantly change their quality of life.”

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“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.

What is Duchenne ​​muscular dystrophy?

DMD is a rare muscle disorder that affects around one in every 3,500 male births globally, according to the National Organization for Rare Disorders (NORD). 

Females are rarely affected.

Symptoms usually become apparent between 3 and 6 years of age. 

 “Treatment will extend to virtually hundreds of boys and significantly change their quality of life.”

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People with this disorder have a mutation in the DMD gene on their X chromosome, which regulates the production of a protein called dystrophin that helps with muscle movement in the body and heart. 

The mutation prevents the creation of this protein, ultimately causing muscle weakness and mobility issues.

To aid in mobility, many children with the disease often rely on braces, canes and wheelchairs.

New gene therapy shows promise

Elevidys, made by Sarepta Therapeutics, Inc. in Cambridge, Massachusetts, is a gene therapy that triggers the production of Elevidys micro-dystrophin, a shortened protein that replaces the dystrophin that is lacking in muscular dystrophy patients, per the FDA’s announcement.

The gene therapy is given as a single-dose injection.

The approval was granted through the FDA’s Accelerated Approval pathway, which fast-tracks access to a drug when there is an “unmet medical need” — and when the medication is likely to relieve patients’ symptoms, improve their quality of life and/or extend their survival.

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The approval was based on data from a two-part study in which one group of 4- to 7-year-olds with DMD was treated with Elevidys and another group received a placebo.

Next, the FDA will require Sarepta Therapeutics to conduct another study to measure whether Elevidys improves physical function and mobility in patients as a means of demonstrating clinical benefit.

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Some potential side effects of Elevidys include nausea, vomiting, fever, low blood platelet count and acute liver injury, the FDA stated. 

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