Google
Trusted Resources: News & Events
Latest announcements and gatherings
An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States
New and life-saving therapy for patients with sickle cell disease (SCD) will soon be within reach. Vertex Pharmaceuticals (collaborating with CRISPR Therapeutics) and bluebird bio have each submitted biologic license applications to the U.S. Food and Drug Administration (FDA) for SCD gene therapy, and a rapid decision is expected. These therapies will join stem cell transplantation as the only curative options for adults with SCD.
A report from the Institute for Clinical and Economic Review predicts that the price of SCD gene therapy will be $2 million per patient (1). Given that more than 50 000 patients with SCD may be …
+myBinderRelated Content
-
APS Scientific Meeting 2019The American Pain Society will hold its ...
-
Sickle Cell Anemia Foundation of OregonThe Sickle Cell Anemia Foundation of Ore...
-
Identifying factors underlying the decision for sickle cell carrier screening among African Americans within middle ...Guidelines recommend that African Americ...
-
Magnesium for treating sickle cell diseaseBACKGROUND: Sickle cell disease is an a...
-
Webinar Series: Sickle Cell Science: Path to Progress – Sickle Cell Disease Care in the Emergency Department: ...To commemorate Sickle Cell Awareness Mon...
-
2016 Annual Sickle Cell Disease Clinical Research MeetingsThe NHLBI Annual Sickle Cell Disease Cli...
-
Early clinical trial data show gene therapy reversing sickle cell anemiaAfter over a decade of preclin...
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.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.