Google
Trusted Resources: News & Events
Latest announcements and gatherings
Sickle Cell Treatment Endari Now Available in the United States
Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.
Sickle cell disease is an inherited blood disorder in which the body produces crescent shaped red blood cells that get caught in blood vessels, ultimately blocking blood flow. An altered form of hemoglobin caused by genetic mutations makes red blood cells fibrous and rigid which forms their abnormal shape.
Sickled red blood cells do not last as long as normal red blood cells, causing anemia to develop in patients. Signs that a patient could have sickle cell disease begin in early childhood and include anemia, repeated infections, and periodic episodes of pain, clinically defined as sickle cell crises.
+myBinderRelated Content
-
Improved clinical outcomes in adults with sickle cell disease and diabetes treated with metforminComplications of sickle cell disease (SC...
-
Boston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
-
Crizanlizumab 5.0 mg/kg increased the time to first on-treatment sickle cell pain crisis (SCPC) and the likelihood o...Background: In the 52-week SUSTAIN stud...
-
New Sickle Cell Disease Research Shows Improved Patient OutcomesEmerging therapies and promising new ins...
-
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene TherapyA 13-year-old boy with sickle cell disea...
-
Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Ga...Acetylon Pharmaceuticals, Inc., the lead...
-
GBT expands sickle cell disease pipeline with worldwide licensing agreement for inclacumab for the treatment of vaso...Global Blood Therapeutics, Inc...
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.