Sarepta Therapeutics has ushered in a new chapter in the treatment of Duchenne Muscular Dystrophy (DMD) with the introduction of ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy approved for this devastating disorder. With the U.S. Food and Drug Administration (FDA) granting accelerated approval in June 2023, ELEVIDYS offers hope by addressing the root cause of DMD....
Corticosteroid therapy is a cornerstone of Duchenne Muscular Dystrophy (DMD) treatment. These medications, such as prednisone and deflazacort, have shown significant benefits in slowing disease progression and prolonging ambulation in individuals with DMD. Corticosteroids work by reducing inflammation and suppressing muscle fiber damage, thus preserving muscle strength and function. However,...
Sarepta’s ELEVIDYS: First Gene Therapy for Duchenne Muscular Dystrophy (DMD) Treatment
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder that primarily affects boys, leading to progressive muscle degeneration and weakness. Historically, DMD has been managed through supportive care, steroids, and emerging pharmaceutical therapies, but there has been no cure. The...
Causes and Mechanism of Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by the absence of dystrophin, a protein that helps keep muscle cells intact. The defective gene that causes DMD is located on the X chromosome, so it primarily affects boys. Girls can be carriers of the defective...
What Does the Future Hold For Gene Therapy in the Duchenne Muscular Dystrophy (DMD) Treatment Market?
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration and weakness. Affecting approximately 1 in 3,500 to 5,000 males globally, DMD is caused by mutations in the dystrophin gene, leading to the absence of dystrophin...
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