Credit: Shutterstock. Zynteglo is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. The Food and ...
The US Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals' CRISPR gene-editing–based therapy exagamglogene autotemcel, or exa-cel, (Casgevy) for individuals aged 12 years or older ...
UCSF Benioff Children’s Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe ...
A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia. The ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...
PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just after her sixth birthday. She lost her appetite and was often doubled over in pain. She continued ...
UCSF Benioff Children's Hospital Oakland provides new therapy to first patient since FDA approval. UCSF Benioff Children's Hospital Oakland is the first hospital in the West to administer a newly ...
While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell transplantation (HSCT) remains the only widely accessible curative option for this ...
Please provide your email address to receive an email when new articles are posted on . NEW ORLEANS — One-time treatment with the gene therapy betibeglogene autotemcel continued to demonstrate ...
UCSF Benioff Children’s Hospital Oakland provides new therapy to first patient since FDA approval. UCSF Benioff Children’s Hospital Oakland is the first hospital in the West to administer a newly ...
Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status. These molecules can indicate if any abnormal processes are taking place, which could point ...
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