DISCLAIMERThe information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.
compose your message
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease
Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.
“Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder,” said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. “Our scientific investments have brought us to a point where we have many more tools available in the battle against sickle cell disease, which presents daily challenges for those living with it. We remain committed to raising the profile of this disease as a public health priority and to approving new therapies that are proven to be safe and effective. Together with improved provider education, patient empowerment, and improved care delivery systems, these newly approved drugs have the potential to immediately impact people living with SCD.”
Sickle cell disease is a lifelong, inherited blood disorder in which red blood cells are abnormally shaped (in a crescent, or “sickle” shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe and chronic inflammation that worsens vaso-occlusive crises during which patients experience episodes of extreme pain and organ damage. Nonclinical studies have demonstrated that Oxbryta inhibits red blood cell sickling, improves red blood cell deformability (ability of a red blood cell to change shape) and improves the blood’s ability to flow.
education & researchUnanticipated consequences identified after implementation of a pediatric emergency department (PED)-based intranasa...Background: The 2014 National Heart Lun...
news & eventsGlobal Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squ...The global sickle cell disease treatment...
news & eventsU.S. Government Awards Missouri Researcher $4.3 Million to Study Sickle Cell in Teens, AdultsThe National Institutes of Health (NIH) ...
people & placesDaniel E. Bauer, MD, PhDDaniel E. Bauer is currently engaged as ...
education & researchTargeted hydroxyurea education after an emergency department visit increases hydroxyurea use in children with sickle...Objective: To evaluate the impact of an...
people & placesUCSF Benioff Children’s Hospital OaklandUCSF Benioff Children's Hospital Oakland...
people & placesNYC Health + HospitalsNYC Health + Hospitals are the largest p...
send a message
Don’t forget to join the oneSCDvoice community!
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.
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.