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November 22, 2022Saving children who are born with medical problems is a significantly important part of healthcare. But there are times when the problems that children have are issues that started when they were still in utero. If the issue isn’t detected then, or it can’t be corrected, that can set the children up for a lot of problems, cause emotional stress for their families and sometimes even come at the cost of their lives at very young ages.
This was the case for Zara and Sara Bashir, both of whom died young from a genetic disorder called Pompe disease. When the parents of these lost children were pregnant again, with Ayla, doctors had a unique plan to save her from the disorder and protect her from the effects it may cause.
The first-ever treatment of its kind infused an enzyme into the umbilical cord when Ayla was still a fetus. This enzyme was crucial for Ayla’s survival, but missing in children who have Pompe disease. Now Ayla is 16 months old, and she’s developing right on schedule. Not only did this treatment save Ayla’s life and spare her parents from the pain of losing another child, but it may pave the way for a number of other treatments that can save children in utero in the future.
Other genetic diseases may also be treatable during the fetal stage, which would save countless lives and also reduce the chances of children needing long-term medical care due to medical conditions related to their genetic makeup. Since the time that Ayla was treated and the research submitted by the doctors who treated her, another fetus has been treated in utero.
The second child was treated for Hunter’s syndrome, which is another genetic issue. The baby was born healthy and is being closely monitored to see whether problems develop as the child grows up. Ayla doesn’t show any signs of Pompe disease, and her parents are very grateful to the doctors and researchers who made that possible.
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