Significant progress has been made in developing gene therapy approaches for Duchenne muscular dystrophy (DMD). This progress marks a crucial milestone in the quest for effective treatments, forty years after the dystrophin gene was cloned. Gene therapy involves modifying or replacing genes to treat diseases, offering hope for addressing the genetic root of DMD.
Advancements in gene therapy, particularly those using adeno-associated viruses (AAV), aim to restore the function of the dystrophin protein. This development represents a hopeful direction in managing and potentially treating DMD more effectively than current treatments. The focus on gene therapy underscores ongoing efforts to improve the lives of those affected by this severe condition (Bengtsson, 2025).
Clinical Trials
This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:
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