In a world first, a fetus has been treated with a really rare genetic disorder that took her siblings’ lives.

The kid, now a toddler, was mentioned in a case study published within the Latest England Journal of Medicine. The bottom-breaking treatment was for a lysosomal storage disorder called Pompe disease.

The physicians successfully treated the kid, Ayla, using a protocol developed on the University of California, San Francisco (UCSF).

“This treatment expands the repertoire of fetal therapies in a latest direction,” co-senior and corresponding creator Tippi MacKenzie, a pediatric surgeon at UCSF Benioff Kid’s Hospitals, said, reported SciTechDaily. “As latest treatments turn out to be available for youngsters with genetic conditions, we’re developing protocols to use them before birth.”

Infantile-onset Pompe disease may cause severe damage to major organs of the body similar to the guts before birth. Physicians at UC San Francisco began enzyme alternative therapy during fetal development to enhance health outcomes for the infant. If left untreated, the disease can turn fatal in early childhood, or result in low muscle tone, or ventilator dependency.

Ayla received six prenatal enzyme alternative treatments at The Ottawa Hospital before being born at term. Currently, at 16 months of age, the kid is undergoing postnatal enzyme therapy at CHEO (a pediatric hospital and research center in Ottawa, Canada) and is doing well.

“After we were having Ayla, we didn’t know if she’d have the option to walk,” Zahid Bashir, Ayla’s dad, said, as per the outlet. “We didn’t know if she’d have the option to speak. We didn’t know if she’d have the option to eat. We didn’t know if she’d have the option to laugh. So, as she hits each of those milestones, we proceed to be amazed at her progress. So, yeah, it’s quite something, that I believe sometimes we may take with no consideration, but more often than not we’re quite aware that she’s a miracle.”

Typically, children born with Pompe disease have enlarged hearts and die inside two years without treatment. Extremely rare, the disease occurs in lower than 1 in 100,000 live births. It’s caused because of genetic mutations that affect the production of an enzyme called acid alpha-glucosidase that breaks down glycogen. The mutation results in the buildup of dangerous levels of glycogen within the body.

“We’ve got been treating our fetal patients using intrauterine therapy for greater than 30 years,” Karen Fung-Kee-Fung, the family’s maternal-fetal medicine specialist on the Ottawa Hospital, said. “The emergence of a latest medical treatment to lift the burden of Pompe disease for this family, and potentially help other families affected by devastating genetic diseases, is each exciting and incredibly satisfying. We feel very privileged and honored to be an element of this international collaboration to assist make this first-in-the-world treatment a reality.”