Emmaus Life Sciences has entered into an agreement to sell the North American rights to its oral sickle cell disease ...
Self-advocacy skills help to build confidence and reduce feelings of helplessness, writes columnist. It's good to start young ...
The answer is "it should be," says columnist. That way, sickle cell patients could receive the support that they need.
Standard treatments can significantly lower the risk of stroke in children with sickle cell anemia, according to a review of ...
Sickle cell disease (SCD) may be cured in some individuals by a stem cell transplant. There also are several approved therapies to help manage the condition and its related complications. Some of the ...
On Dec. 8, the U.S. Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), a groundbreaking decision that now offers patients more than one ...
The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both ...
For most of his 39 years, Charles Hough lived with extreme pain caused by sickle cell disease, which dramatically reduced blood flow throughout his body. But thanks to the gene therapy he received in ...
A newly designed viral vector — the vehicle that delivers a gene therapy to a patient’s cells — for use in sickle cell anemia is more efficient than earlier vectors at introducing healthy copies of ...
Using tocilizumab off-label in four young adults with sickle cell disease (SCD) who developed hyperhemolysis syndrome as a complication of a blood transfusion stopped the premature destruction of red ...
The Phase 1/2 CLIMB-SCD-121 trial (NCT03745287) is currently evaluating the safety and effectiveness of a single infusion of CTX001 in adults up to age 35 with severe SCD. Two patients have been dosed ...
The investigational gene-editing therapy OTQ923 resulted in normalization of the speed of blood flow in the brain of three adults with sickle cell disease (SCD), according to data from a Phase 1 ...
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