Amy Cohen is running the New York City Marathon despite living with the painful effects of sickle cell disease.
A one-time gene-editing therapy, exagamglogene autotemcel (exa-cel), has demonstrated sustained and clinically meaningful ...
Vertex Pharmaceuticals Inc. (($VRTX)) announced an update on their ongoing clinical study. Vertex Pharmaceuticals Inc. is conducting a Phase 3 ...
The treatment of sickle cell disease is on the cusp of a historic breakthrough, with makers of two gene-based treatments for the debilitating blood disorder hoping for regulatory approval this year.
Patients with sickle cell disease and beta-thalassemia treated with exagamglogene autotemcel reported substantial and ...
Modern medicine is one step closer to potentially curing sickle cell disease. On the heels of the U.K.'s authorizing the world’s first gene-edited treatment for sickle cell disease on Nov. 16, the U.S ...
In a landmark decision, the Food and Drug Administration Friday approved the first gene-editing treatment to alleviate human illness. The FDA approved two gene therapies for anyone 12 and older ...
Real Science on MSN
The Blood Disorder That Gene Editing Finally Began to Cure
In 2024, regulators approved Casgevy, the first CRISPR-based therapy to treat sickle cell disease and beta-thalassemia — a ...
Cristina Pina receives funding from Children's Cancer and Leukaemia Group via the Little Princess Trust and the National Centre for the Replacement, Reduction and Refinement of Animals in Research.
WRIC Richmond on MSN
5-year-old cured of sickle cell after life-saving bone marrow transplant
After four years of battling a severe blood disease, five-year-old Zion Ortiz is finally free of sickle cell anemia.
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