Progressive muscle weakness and eventual mortality as a result of cardiomyopathy or respiratory complications are the hallmarks of Duchenne muscular dystrophy (DMD), a severe X-linked disorder. At present, there is no cure for DMD, and the primary focus of standard treatments is on symptom management. Gene therapies can more effectively address the genetic cause of the disease by employing immunosuppressive measures and optimized vector designs.
What are the effects of gene therapy treatments in Duchenne muscular dystrophy (DMD) disease, do gene therapies work? Here is a comprehensive analysis.
