Hemgenix (etranacogene dezaparvovec-drlb) is a prescription gene therapy approved for the treatment of adults with hemophilia B, a rare genetic bleeding disorder caused by factor IX deficiency. Unlike traditional treatments...
Haegarda is a prescription therapy designed to help people with hereditary angioedema (HAE), a rare genetic condition that causes sudden, painful swelling attacks in the body. Its active ingredient is...
Elevidys (delandistrogene moxeparvovec-rokl) is a pioneering gene therapy developed to treat children with Duchenne Muscular Dystrophy (DMD)—a rare, progressive, and ultimately fatal genetic disorder. This FDA-approved therapy represents a new...