Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Gene Therapy: What You Need to Know (Warrior FAQs)
source: Sickle Cell Disease Association of America Inc.
year: 2023
summary/abstract:Two gene therapies were recently approved by the Food and Drug Administration (FDA) to treat sickle cell disease: Casegevy from CRISPR/Vertex and Lyfgenia from bluebird bio. You probably have questions about these new treatment options. Read more below.
Is gene therapy a cure for sickle cell disease?
Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. It causes a big decline in pain episodes, but we need to learn more about long-term impacts and side effects. It is also not a “one-and-done” treatment. The FDA currently recommends 15 years of patient follow up.
Related Content
-
Sickle Cell Foundation of Georgia, Inc.Since 1971, the Sickle Cell Foundation o...
-
Inspiring More MindsInspiring More Minds, Inc. is dedicated ...
-
FDA grants CRISPR gene therapy fast track designation for sickle cell diseaseThe FDA granted fast track designation t...
-
Sulzner Sickle Cell Anaemia FoundationSulzner Sickle Cell Anaemia Foundation i...
-
Karen Proudford, PhDDr. Karen Proudford is President of the ...
-
KNOWvember Webinar: A grandparent’s voice – Advocacy and support for chronic pain familiesWhile parents are often the prim...
-
Gary A. GibsonGary A. Gibson has accumulated forty yea...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.