Ada Gonzalez lives with sickle cell disease and enrolled in a clinical study decades ago that she benefits from today.
Jewel is a teenager living with sickle cell disease. She and her mother, Paula make informed decision about participating in clinical studies.
To determine whether having sickle cell trait (SCT) is a risk factor bone thinning at an earlier age and to understand why individuals with sickle cell disease (SCD) have thinner bones than healthy individuals do.
1 US site
18 to 45 years
SCD and SCT
February 22, 2020
LentiGlobin gene therapy led to substantial reductions of sickle hemoglobin and considerable improvement in key markers of hemolysis.
December 25, 2019
Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped — producing a protein that could alleviate the worst complications of sickle cell.
November 25, 2019
With Oxbryta, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction. This therapy provides a new treatment option for patients with this serious and life-threatening condition.
November 19, 2019
Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.
Sending updates featuring new studies and other relevant information about emerging sickle cell treatment options.