Care hurdles now add to the disease burden faced by U.S. sickle cell patients, despite the promise of emerging options like gene therapy.
A study finds sickle cell trait in babies is linked to distinct molecular markers detectable within the first week after birth.
Asthma is about seven times more likely in children with sickle cell disease than in their unaffected siblings, according to ...
Sickle cell disease (SCD) is an inherited disorder caused by a mutation in the gene encoding a subunit of hemoglobin, the protein that carries oxygen in red blood cells. Mutant hemoglobin — called ...
Sickle cell anemia is an inherited blood disorder. It is caused by a mutation in the gene encoding for hemoglobin, the protein in red blood cells that binds to oxygen so it can be transported ...
A cystatin C–based blood test may provide a more reliable way to monitor kidney function in children and young adults with SCD, a study found.
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 ...
What is a blood transfusion? A blood transfusion is a medical procedure through which healthy blood cells or blood components are transferred from a healthy donor to someone in need. It may be used to ...
The first participant with sickle cell disease (SCD) has been dosed in a clinical trial of HBI-002, an experimental, low-dose, oral liquid formulation of carbon monoxide being developed by Hillhurst ...
The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted ...
What is Casgevy for sickle cell disease? Casgevy (exagamglogene autotemcel), formerly known as CTX001 or exa-cel, is an approved gene-editing therapy used for people with sickle cell disease (SCD) who ...
What is Endari for sickle cell disease? Endari (L-glutamine) is an oral therapy approved in the U.S. for children and adults with sickle cell disease (SCD), ages 5 and older. It’s used to reduce acute ...
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