Sickle cell disease, while rare, is the most common inherited blood disorder and affects over 100,000 people in the United States, more than 90% of whom are Black according to the Centers for Disease Control and Prevention. ...
Jul 31, 2024
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Scientists have created the first bioengineered bone marrow model that can support the type of human stem cells that are crucial for bone marrow transplants and in vitro study work.
Jul 17, 2024
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A large team of medical researchers at Novartis Biomedical Research has developed a possible treatment for people with sickle cell disease. In their project, published in the journal Science, they conducted an exhaustive ...
New research led by UCLA Health has found a drug that treats insomnia works to prevent the addictive effects of the morphine opioids in mice while still providing effective pain relief.
Jul 9, 2024
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Roughly 50 families scattered across the world share ultra-rare variants in a particular gene. Silent for years, the inherited mutations make themselves known when patients reach the fourth decade of life. Changes in vision ...
Jun 17, 2024
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By completely or even partially depleting a protein called midnolin in B cells, UT Southwestern Medical Center researchers suppressed leukemia and lymphoma in a mouse model genetically prone to these cancers.
May 15, 2024
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In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) ...
Apr 25, 2024
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A new study led by Indiana University School of Medicine researchers has found that a brain network condition called "explosive synchronization" could be the cause of extreme pain crises in people with sickle cell disease.
Apr 24, 2024
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A drug approved to treat pulmonary arterial hypertension may be effective at managing hypertension and end-organ damage in patients with sickle cell disease, according to a new study published in Lancet Haematology.
Apr 11, 2024
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A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher of a trial published in the New England Journal ...
Feb 2, 2024
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