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Children’s Wisconsin is now offering the newly FDA-approved Casgevy therapy
The MACC Fund Center for Cancer and Blood Disorders at Children’s Wisconsin is now offering the newly FDA-approved Casgevy therapy to children 12 years and older who have severe sickle cell disease or transfusion-dependent thalassemia.
Casgevy is made from the patient’s own blood stem cells, which are modified using CRISPR technology, and returned to the patient in a single-dose infusion as part of a stem cell transplant. Prior to the infusion, the patient’s stem cells are collected for the modification, and then the patient goes through a round of high-dose chemotherapy intended to destroy the existing cells from the patient’s bone marrow that make the mal-formed blood cells. Gene therapy is considered a “potentially curative” or “transformative therapy.”
Casgevy Benefits:
• 93 percent of patients having at least 12 consecutive months without a severe vaso-occlusive crisis
• 92 percent of patients with thalassemia were transfusion-free for more than 12 months
• Results are achieved with significantly less serious side effects than existing therapies
For additional information click here Children's Wisconsin Macc Fund Center Sickle Cell Gene Therapy