Faith – Krabbe Disease, a Devastating Genetic Nerve Disorder

Faith's Patient Story

Baby Faith has a chance to lead a full life — thanks to her older sister, Zarley, and timely treatment of Krabbe disease.

Both sisters were born with Krabbe disease, a rare degenerative nerve disease.

Zarley’s diagnosis came too late to save her life. She died eight years before Faith was born.

But, because of Zarley, her parents knew how to give Faith every chance for a full life after hearing the prenatal diagnosis.

A Move from L.A. to Pittsburgh for Expert Care

The family left their home near Los Angeles and moved to Pittsburgh for Faith’s treatment.

They expressly sought care from Maria Escolar, MD, at the Center for Rare Disease Therapy at Children’s Hospital of Pittsburgh of UPMC.

Dr. Escolar is an internationally renowned expert on leukodystrophies — a group of rare genetic diseases.

Faith's Cord Blood Transplant at 23 Days Old

At 23 days old, Faith became one of the youngest patients ever to receive an umbilical cord blood transplant at Children’s Hospital.

A cord blood transplant involves weeks of hospitalization followed by months of close follow-up care.

But, just 13 days after Faith’s transplant, blood tests showed that she had fully engrafted. This means Dr. Escolar found that 100 percent of the blood cells in Faith's body formed from the donated cord blood.

Doctors expect that Faith will be well enough to stop taking immunosuppressant medication by May — nine months after her transplant. She and her mother will then return to Pittsburgh — up to four times a year — for follow-up visits with Dr. Escolar.

Whatever the future holds for Faith, her mother has a sense of comfort. She knows that her daughter is receiving care from doctors with a wealth of knowledge treating children with Krabbe disease.