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Understanding the Importance of Gene Therapy for Rare Diseases

Gene therapy is a type of medical treatment that involves adding, removing, or changing a person’s genetic material—also known as their DNA. Gene therapies are being studied in patients with serious or life-threatening rare diseases because they focus on correcting the root cause of the disease rather than just treating the symptoms. As a result, gene therapies offer patients a better chance at long-term improvements in how their bodies function and their quality of life. Several gene therapies have been approved by the U.S. Food and Drug Administration (FDA) and many others are currently being evaluated in clinical trials. 

NORD has developed a variety of educational resources about gene therapy to help patients, families, healthcare providers and the public learn about this emerging therapeutic area including the basics of gene therapy, how it is currently used and its potential for future therapies for rare diseases.  

Get started by downloading NORD’s gene therapy fact sheet or listening to NORD’s podcast, Ungeeking The Speak: Dr Rachel Bailey Talks “Gene Therapy 101”.

Download fact sheet