Fetal Therapy Treats Genetic Disorder In-Utero

In-utero fetal therapy, a new treatment for babies with rare genetic diseases, successfully treated a child with Pompe disease, and there is hope that other genetic diseases can be cured with this innovative therapy. “Pompe disease is just one of a group of glycogen storage diseases, and most cause severe disease and often progress to death of the child at very young ages,” said Alan Fishman, M.D., specialty medical officer for maternal-fetal medicine at Pediatrix® Medical Group. Dr. Fishman said theoretically, any glycogen storage disease could be treated in the same ways as in the child with Pompe disease. “The challenge is to characterize the individuals who are at risk and to correctly identify those likely to be severely affected at a young age,” he explained. “In this particular family, this was clear from the family history.”    

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