Drug Class
gene therapy
Type
brand
Active Ingredient
delandistrogene moxeparvovec
📋Overview
Elevidys is a gene therapy medication designed for certain individuals with Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle weakness and degeneration. It works by delivering a functional gene to help produce a form of dystrophin, a protein essential for healthy muscle function. Elevidys is associated with efforts to address the underlying genetic cause of DMD, particularly in patients with specific genetic profiles. Its use represents an innovative approach in the management of inherited neuromuscular diseases.
📚References
Information from MedlinePlus.gov (NIH).
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