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STRONG SCD
The STRONG SCD study is evaluating different dose levels of an oral investigational drug designed to lessen SCD complications, including pain crises.
30 US sites, 1 Lebanon site
16 to 70 years
SS, SC, Sβ0 thal, Sβ+ thal
Phase 2
SCTaware
This is a study for parents of infants with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in-person SCT education at the Institution.
1 US site
18 to 65 years
AS (sickle cell trait)
Phase 1 / Phase 2
Sickle Cell and Bone Health
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
N/A
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.