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
Bluebird Bio Presents Data From LentiGlobin Gene Therapy Trial
Bluebird Bio’s investigational gene therapy LentiGlobin safely prevents serious vaso-occlusive crises and acute chest syndrome in adolescents and adults with severe sickle cell disease (SCD), according to recent data from a Phase 1/2 clinical trial.
The findings, “Outcomes in patients treated with LentiGlobin for sickle cell disease (SCD) gene therapy: updated results from the Phase 1/2 HGB-206 group C study,” were recently presented at the virtual 25th European Hematology Association (EHA) Annual Congress.
“The promising results of this study, which show patients have an almost complete elimination of VOCs [vaso-occlusive crises] and ACS [acute chest syndrome], suggest LentiGlobin … has real potential to provide a significant impact for people living with sickle cell disease,” Julie Kanter, MD, said in a press release.
news & eventsCalifornia’s Stem Cell Agency Invests in Stem Cell-Based Therapies Targeting Sickle Cell Disease and CancerThe California Institute for Regenerativ...
education & researchOrphan Drugs for Sickle Vaso-occlusion: Dawn of a New Era of Targeted TreatmentWhile an orphan disease in the USA, sick...
news & eventsEarly clinical trial data show gene therapy reversing sickle cell anemiaAfter over a decade of preclin...
news & eventsNew Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Cr...SANGUINATE™, the only investigational ...
education & researchRandomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased ...Treatment of vaso-occlusive crises (VOC)...
news & eventsBoston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
news & eventsVoxelotor can Promote Long-Term Benefits in Teens, Adults With SCD, Latest Clinical Data ShowGlobal Blood Therapeutics’ lead therap...
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.