People with sickle cell disease who receive nonmyeloablative stem cell transplants are at greater risk for developing new tumors, according to a new study published in the Journal of Clinical Oncology.
Mar 9, 2023
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New educational materials to help the sickle cell disease community learn about gene therapies for the disease are now available from researchers at the National Human Genome Research Institute (NHGRI), part of the National ...
Sep 28, 2022
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Bluebird Bio Inc.'s gene therapy for a brain-wasting disease received accelerated approval from U.S. regulators, making it the first treatment of its kind for children living with cerebral adrenoleukodystrophy.
Sep 19, 2022
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In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children's Hospital of Philadelphia (CHOP) presented new data on an investigational therapy ...
Jun 14, 2022
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In a study published today in Blood Advances, researchers found that privately insured individuals with sickle cell disease (SCD) spend approximately $1.7 million on disease-related medical expenses over their lifetime. These ...
May 16, 2022
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Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and delayed growth or puberty. Treatment ...
Feb 16, 2022
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Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored ...
Feb 15, 2022
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A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.
Dec 13, 2021
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Sickle cell disease, which is a group of inherited red blood cell disorders, affects approximately 100,000 people in the U.S., according to the Centers for Disease Control and Prevention.
Sep 10, 2021
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Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
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