Sarepta's ELEVIDYS is the first FDA-approved gene therapy for Duchenne muscular dystrophy
Sarepta Therapeutics is a biotechnology company focused on the development of precision genetic medicine for rare diseases. The company's three main areas of focus are gene therapy, RNA-targeted exon skipping, and gene editing.
Gene therapy involves the use of genetic material to treat or prevent a disease. Sarepta is developing gene therapies for a variety of rare diseases, including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophy (LGMD), and Pompe disease.
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness and loss of muscle mass.
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness and loss of muscle mass. It is caused by a mutation in the dystrophin gene, which leads to the absence or deficiency of the dystrophin protein. Dystrophin is a key protein that helps to maintain the integrity of muscle cells. Without dystrophin, muscle cells are more susceptible to damage and eventually die. DMD primarily affects boys, with symptoms typically appearing in early childhood. The disease progresses rapidly, and individuals with DMD typically lose the ability to walk by their teenage years. DMD is a life-threatening condition, and most individuals with the disease do not survive beyond their early twenties. There is currently no cure for DMD, but there are a number of treatments that can help to slow the progression of the disease and improve quality of life.
ELEVIDYS is the first FDA-approved gene therapy for Duchenne muscular dystrophy.
ELEVIDYS is the first FDA-approved gene therapy for Duchenne muscular dystrophy. It is a one-time gene therapy that is delivered intravenously. The therapy works by delivering a gene that codes for a shortened form of dystrophin, a protein that is missing or defective in people with DMD.
The FDA's approval of ELEVIDYS is based on the results of two clinical trials. In the first trial, ELEVIDYS was shown to increase the expression of dystrophin in skeletal muscle. In the second trial, ELEVIDYS was shown to improve motor function in patients with DMD.
The most common side effects of ELEVIDYS were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia.Sarepta Therapeutics has committed to completing a confirmatory trial to verify the clinical benefit of ELEVIDYS. The trial is expected to be completed in late 2023.
ELEVIDYS is the first FDA-approved gene therapy for Duchenne muscular dystrophy and has the potential to make a significant difference in the lives of patients by addressing the root genetic cause of the disease and providing the potential to alter the trajectory of this degenerative disease. 
