Center for Rare Disease Therapy Patient Stories

Learn how families are finding help and hope through the expertise of the Center for Rare Disease Therapy at Children’s Hospital of Pittsburgh of UPMC. Watch their stories below.

Levi suffered from Krabbe Disease. His family traveled from his home in Australia to receive care at Children’s Hospital of Pittsburgh of UPMC. Learn more about his care.
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Lily suffered from Krabbe Disease. Read about the success of her procedure and experience at Children's Hospital Pittsburgh of UPMC.
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Ally suffered Combined Immune Deficiency. Read about the success of her procedure and experience at Children's Hospital Pittsburgh of UPMC.
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Grace suffered from Steroid Resistant Nephrotic Syndrome. Read about the success of her procedure and experience at UPMC.
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Joshua suffered from Glycogen Storage Disease. Read more about the success of his procedure at Children's Hospital of Pittsburgh of UPMC.
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Leah suffered from Maple Syrup Urine Disease. Read about her experience at Children’s Hospital of Pittsburgh of UPMC.
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