Gene Therapy Cure for Beta Thalassemia
Nine-year-old Ada is the very first patient on the West Coast to receive a life-changing curative therapy for beta thalassemia, a rare blood disorder. UCSF Benioff Children’s Hospital Oakland led clinical trials of this innovative therapy, which recently received FDA approval. Now 90% of patients, including Ada, are free from monthly six-hour blood transfusions to manage their blood disorder.
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