ELEVIDYS

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Gene therapy: Following Pfizer’s Unexpected Failure, Sarepta is celebrating its much-awaited full approval! Sarepta’s stock price has shot up over 32%

Duchenne muscular dystrophy (DMD) is a severe genetic disorder primarily affecting young boys between the ages of two and three. As per Delveinsight’s estimates, there were approximately 16K DMD patients in 2023 in the United States, with ˜75% of the patients below the age of 15. This condition leads to progressive...

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Sarepta’s ELEVIDYS: First Gene Therapy for Duchenne Muscular Dystrophy (DMD) Treatment

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...

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