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
-
Lashawn HutchinsonLashawn Hutchinson is CEO at Shawn's Tes...
-
Thalassaemia & Sickle Cell Society of AustraliaThe Thalassaemia and Sickle Cell Society...
-
FDA grants CRISPR gene therapy fast track designation for sickle cell diseaseThe FDA granted fast track designation t...
-
Joining hands: SCDAA and Emmaus medical partner to fight sickle cell diseaseIn observance of World Sickle Ce...
-
Today’s Faces of Sickle Cell Disease: Jennifer NsenkyireJennifer Nsenkyire, who is originally fr...
-
New Hope for Our Youth: 2021 Indiana Sickle Cell Conference – Virtual EditionFriday, April 23, 2021 8:30am – 12:30...
-
Prodigy’s death shines light on slow progress against sickle cell diseaseThe death of the rap artist Prodigy (Alb...
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.