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 approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe anemia, with a need for lifelong blood transfusions and the risk of fatal organ damage.
"This new therapy could be a life-changer for beta thalassemia patients, who have required cumbersome and expensive, lifelong monthly blood transfusions," said Mark Walters, MD, director of the pediatric Bone Marrow Transplant program at UCSF Benioff Oakland and chief of pediatric hematology at UCSF, who led the Oakland clinical trial.
Related StoriesIt is normally detected by newborn screening, with treatment beginning in infants as young as 6 months old to overcome having too few red blood cells to sustain life. Patients also require regular treatment to reduce high levels of iron that result from the transfusions, potentially impacting the function of their heart, liver, and endocrine system organs like the pancreas and pituitary gland, which are needed for growth and development.
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