The Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act of 2018 (S. 2465) which reauthorizes a sickle cell disease prevention and treatment program and to provide grants for research, surveillance, prevention, and treatment of heritable blood disorders. To learn more click here.
Latest News and Research
Sickle Cell Disease Treatment Act of 2018 Law
60 Minutes Showcases Upcoming Gene Therapy
There are many gene therapy treatments in the works for individuals with sickle cell disease. One initiative by NIH, provided a segment on 60 minutes of its current research. To learn more click here.
What are the newest sickle cell disease trials?
To learn more about sickle cell disease trials, please click here.
FDA Approves Endari for Sickle Cell Patients
After more than 20 years without any new medication specifically for sickle cell disease, the FDA approves Endari to Emmaus Medical Inc in 2017. During a randomized trial, patients who received Endari reported less pain, less acute chest syndrome and had reduced hospitalizations. To learn more click here.
Oxbryta (Voxelotor) Approved for Sickle Cell Patients
Oxbryta (Voxelotor) was approved by the FDA in November 2019. Learn more about this treatment, prescription information and patient information line here.
Adakveo Approved for Sickle Cell Patients
Adakveo was approved by the FDA in November 2019. Learn more about this treatment, prescription information and patient information line here.
Are there any sickle cell trait trials?
To learn more about sickle cell trait and any upcoming trials click here.
First U.S. Patient receives CRISPR therapy
CRISPR uses its gene therapy in the first US patient. To learn more click here.