Gene Therapy Offered This 7-Year-Old Freedom. The Price: A Grueling Year
December 13, 2023
(STAT News) – Fewer than 15 other people with beta thal have started the Zynteglo process, but gene therapies for the far more common sickle cell disease, from Bluebird and from Vertex and CRISPR Therapeutics, were approved in the U.S. last week. Treatments for immune diseases and cystine buildup are on the way as well. All of these gene therapies will require stem cell transplants like Shelby’s, meaning thousands more patients will go through the same arduous process in the near future.
In chronicling Shelby’s journey, STAT visited her in the hospital and spent more than a year talking to the Campbell family, other people with beta thalassemia, and those who treat it to understand how this kind of slow-moving miracle affects patients and caregivers during and after the therapy. (Read More)